Crispr-Cas9 is the breakthrough in genome engineering that just changed every aspect of our world, and will lead to its end ?

[Uhohinc]

https://www.youtube.com/watch?v=SuAxDVBt7kQ No drug company will be safe with its market when this becomes widespread.If you did not like Monsanto and its rudimentary genetic modification of seeds, this will be a watershed moment for humans.  This will transcend international politics, ways to kill on mass scale, designer babies, animal slavery, a real Jurasic Park..............possabioilties are endless. 

[MrPoonz1]

Things were depressing already what with the share price and all, oh well, we have to get to the bottom before we get to the top eh? Two months to go until ecstasy or oblivion....

[Uhohinc]

The researchers behind 'the biggest biotech discovery of the century' found it by accident

• Kevin Loria

• Jul. 7, 2015, 2:14 PM
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Peter Barreras/Invision/APBreakthrough Prize life science laureates Emmanuelle Charpentier and Jennifer A. Doudna arrive at the 2nd Annual Breakthrough Prize Award Ceremony at the NASA Ames Research Center on Sunday, November 9, 2014 in Mountain View, California.

When Jennifer Doudna and Emmanuelle Charpentier discovered the most powerful DNA editing technology we know of, CRISPR-Cas9 — something MIT Technology Review described as "the biggest biotech discovery of the century" — they weren't looking for a world-transforming DNA editing tool.

Doudna tells Business Insider that they were studying the system that bacteria use to defend themselves against viruses, something that might be considered basic research, science pursued just for the sake of greater understanding.

Their project was a curiosity-driven initiative to understand a biological mechanism, not an attempt to create or uncover something immediately useful.

But then: "One day ... we realized, gosh, this could be a very powerful technology," says Doudna.

They noticed that the system bacteria use to shut down viruses had an uncanny way of targeting specific sections of virus DNA — and that, with the correct programming, this system could seek out any section of DNA and slice it up. Not only that, if accompanied by other coding material, this process could also replace one section of DNA with a new section of DNA.

They realized they'd found an incredibly precise tool.

We've had gene-editing technology for decades, but now "we're basically able to have a molecular scalpel for genomes," says Doudna, a biologist at the University of California at Berkeley. "All the technologies in the past were sort of like sledgehammers."

In the hands of genetic engineers, having an accurate way to edit genomes composed of millions of letters is incredibly exciting.

"It's really going to just empower us to have more creativity ... to get into the sandbox and have more control over what you build," says Dustin Rubinstein, the head of a lab working with CRISPR and other genetic engineering tools at the University of Wisconsin–Madison. "You're only limited by your imagination."

This is a hugely exciting advance, one that could transform everything from human health to energy production. Genetically modified creatures and designer babies are just a few of the seemingly sci-fi ideas that now appear to be closer to reality, or at least a possibility (there are, of course, serious ethical issues to resolve first).

This discovery — as an unplanned result of what Doudna and Charpentier were working on — could be called a scientific accident, and one of the most extraordinary ones in history.

The two scientists realized the potential applications for the system they were studying and then conducted experiments to show how it worked, which required both flashes of insight and a great deal of expertise. Yet still, "it's serendipitous work — there's an element of luck involved," says Doudna.

For that reason, she says this discovery highlights the importance of funding basic research, as that is the sort of work that can advance knowledge that then — unexpectedly — can lead to a transformative practical discovery.

Funding for this type of work has fallen significantly over the past 50 years, according to a recent report out of MIT. Here's a chart showing the drop in US funding for basic research from 1968 to 2015:

MIT

"[Supporting basic research] is something that the US has done in the past," says Doudna. "I think we're in danger of losing that right now."

SEE ALSO: The age of genetically engineered animals has arrived

NOW WATCH: 9 facts about the brain that will blow your mind

Read more: http://www.businessinsider.com/the-people-who-discovered-the-most-powerful-genetic-engineering-tool-we-know-found-it-by-accident-2015-6#ixzz3ghHji3cY

[Uhohinc]

I do not know how long it will take, but its coming. I thought we had at least a decade before someone figured out how to do not just editing out at a section of genes, but then changing them.   It may take a little more breakthrough knowledge to use this with stem cells.

I think the goverments of the world, as well as the private investors are raising money for this already. As with Craig Ventners six hundred million deal with Exxon to create a bacteria or yeast that byproducts a oil hydrocarbon to take the place of oil drilled from the ground. He did create lab life last year, but this Crispr-cas9 will make it able for anyone in thousands of labs to do. In place of writing computer code, the new world will be writing dna code.  Wait til that N. Korean dictator gets into making his own monster mammals or insects.

A couple of months ago, a US lab affiliated to Fort Deitrich, Maryland in error mailed some anthrax microbe to about 15 labs across USA and by mistake some of the anthrax was still alive, capable spores. Anthrax is present in soil, and from time to time spores kicked up or in a wind can enter a cows lungs. It takes more than a few spores to overwhelm the immune system, and then it multiplys. Now why would the USA want to research anthrax, it has no useful human purpose other than to kill. Or they know it is so successful as a bioweapon that they are trying to research a anti-body for it.

But now with this Crispr-cas9 this could make the Anthrax very deadly. 

The good is such that, for example, the enzyme in a blood cell of an EPP  which dose not allow the proper creation with the heme protein for biosynthesis of such enzyme can easily be taken out and then the proper genetic code as in a normal human can be inserted in place. Then this cell can be multiplied and put back into the marrow to make proper blood cells.  There still needs to be better research on the stem cell end here, but it appears to be successful in doing this type of stem cell in the last year in humans.

As for food, what has taken five thousand years of farmers taking the best attributes of seeds and cross hybridization or breeding your meaty and fat chicken to a like rooster will be done immediately. Or even a orange tree that grows in England or New York that has oranges the size of watermelons, on a tree that produces stronger limbs, uses almost no water, needs almost no sunlight, and produces on one tree what a hundred trees today do.

[coolje...@hotmail.com]

Great info, Uho. Wish we had a "thank you " button here :)

Taking this breakthrough one step further ( into an arena I at least know SOMETHING about), eventually the missing "c" in braca 1& 2's might be genetically reinserted? Perhaps at birth? Or at any point in life?

Scenesse has always held this metaphorical vision in my mind as "filling in the blanks" wherever the body is missing a crucial element, I.e. EPP.....

Exciting times indeed.

[Uhohinc]

I think it is clear that the "editing" of dna to a zygote, (fertilized human egg as it starts from being the one and first human cell which will then divide trillions of times, and specialize and position  to make a human) ability is already here. Just very difficult and expensive. This Crispr-Cas9 brings down the difficulty and cost tremendously.

 As for editing the genes in a full grown human there appear to be more difficulty but not without some success. It looks like the Crispr-Cas9 was just done successfully in Cystic Fibrosis, and I think in Huntingtons Disease. Both single gene disorders. With the cost of ones own personal genome copied on a CD rom coming down so fast the possibilities are profound in the nearing future.  With a genetic disorder as EPP and several hundreds disorders like it, it is a known  gene, or just a couple genes that cause a defective protein, or not making enough of that dna's protein, or making to much of that protein. Essentially the basis of all genetic disease.

 With something like Vitiligo it is not known the one true cause or gene(s). Probably not the melanocyte, but the immune cell that attacks it. No one knows which gene or interaction of genes that could be changed to stop it. Almost all the researchers involved in Clinuvel's vitliligo research, are also involved in the area of  harvesting of melanocytes cells research for "cure" of vitiligo. They are shaving off the healthy melanocytes, and transplanting them.

 There will be several technologies I think that will avail to make it simple and cost efficient to proficiently change the dna in a adult human. This area needs breakthroughs, but they are happening.

 For decades, the research has attempted to use a virus to piggy back into the cell nuclei but with problems. This Crispr-Cas9 appears to be "luck" and a "mistake" according to the two ladies at UC Berkley whom discovered it. There are already legal implications and I think liars out there faking their reseach log books to claim it as theirs first.

I think you have a couple of the Fry's Electronics megastores in Bay area. The one near L.A. has a display of a 3D printer operating in a plastic case.  It is sold as a kit for a couple thousand dollars. Very early technology, but it has the raw plastic in different colors feeding into similar to a printer cartridge as we all see on our computer two dimensional printers on paper.  With the 3D printer you can take a picture of something and "print " it as an object. There are more sophisticated ones now that are not using ink, or plastic, but are using cells to "print". I saw a tease link on the internet where they are making a new face for a little girl with a 3d printer. Lost your finger in a blender, no problem just print a new one. Need a new heart, or kidneys, print it. This is not sci fiction now, it is being perfected.

To think "out there" now, With the dna of all living things based on just four simple amino acids. Now think of just four "printer cartridges" with just these amino acids. One could envisage a computer hacker or hobbyist going on their computer and manipulating or creating anything, something, ?

[Uhohinc]

http://drmicozzi.com/gene-therapy-is-still-a-bust-for-most-diseases

A dissenting opinion. It may be befor the realization of Crisp-Cas9

[Uhohinc]

June 30, 2015

Experts at MDI Bio Lab conference see future therapeutics in regenerative medicine

PHOTO / Rogier van Bakel / eagereyephoto.com

Dustin Shillcox (right) with researcher Claudia Angeli. Shillcox has relearned how to stand using an experimental treatment from Angeli called spinal cord epidural stimulation.

by Lori Valigra

Dustin Shillcox sat in his wheelchair before an audience of scientists last Friday evening, asked everyone to stand up and then said, "How did you stand up?"

Greeted by silence, he noted that people normally don't think about how they move. But Shillcox, 31, has had to consciously relearn how to stand. He lost movement from his chest down after a 2010 driving accident in which he was ejected 80 feet through his car window.

Shillcox is using an experimental treatment called spinal cord epidural stimulation, which involves implanting an array of electrodes into his spine that are helping his brain learn how to walk again. The treatment is part of the growing field of regenerative medicine, which also includes stem cell transplants, gene editing and other experimental techniques to essentially undo the damage caused by aging or injury. While much of the research still is early stage and being done in animals, some, like spinal cord epidural stimulation, already are being tested in people like Shillcox.

The promise of regeneration

"Some venture capitalists are investing in long-term projects that have a specific regenerative medicine focus," said Dr. Thomas Rando of Stanford University School of Medicine in Palo Alto, Calif. He was among a group of regenerative medicine researchers at the REGEN2015 meeting from June 26-28 at the MDI Biological Laboratory in Bar Harbor. The lab specializes in regenerative medicine.

"I think regenerative medicine has a great future in therapeutic development," added Eric Olson, Ph.D., professor of molecular biology at the University of Texas Southwestern Medical Center in Dallas. He also predicted that gene editing — where one letter of the complex genetic alphabet comprising DNA is changed to fix a mutation — will be used to treat a disease within a decade.

Most of the researchers are working on basic animals, like worms, zebrafish and mice, to first find out how they are able to regrow severed limbs and heal from severe wounds. Humans also are believed to have such recovery mechanisms in their bodies, but so far they only work in organs like the liver and certain kidney tubules.

MDIBL itself in 2013 launched the first company in its 115-year history, Novo Biosciences Inc., to study an experimental drug that has been shown to double the regrowth rate of tissue after amputation using a zebrafish.The company is studying the drug in other animals to see its effect on damaged heart tissue, with the hope of one day using it in humans.

Still early stage

And while regenerative medicine still is in its early stages, it gives hope to patients like Shillcox. He said the stimulator, which he turns on and off by remote control, and can use only four hours a day, not only helped him to stand using a walker. It also helped regulate his blood pressure, increase his lung capacity and rebuild the muscle mass he lost from sitting all day.

So far, four patients have received the implant at the Kentucky Spinal Cord Injury Research Center, said Claudia Angeli, Ph.D., an assistant professor at the center. She said she intends to use the implant in four more patients, three of whom have already been selected for the experimental treatment.

The implant stimulates the spine, but researchers think that the brain may be reacting to visual and sound cues and actually building new nerve pathways around the damaged spine to the legs. The more the patient practices moving his or her legs, the better the movement.

Angeli said the hope is one day to have the procedure used more widely outside of an experimental setting. But she and her colleagues still are looking at the optimum configuration of the electrodes for each specific patient.

"My goal and my challenge are to walk again," said Shillcox. "But first I have to stand."

Read more about Shillcox's story on MDIBL's website.

Read more

MDI Bio Lab gets $392K for disease research

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[Uhohinc]

Cdk12 is the main gene control of cell cycle arrest, which must take place for dna repair.  Scenesse does this.

Engineering an analog-sensitive CDK12 cell line using CRISPR/Cas

• Bartlomiej Bartkowiak,
• Christopher Yan,
• Arno L. Greenleaf^,

• Department of Biochemistry, Duke University Medical Center, United States

Received 21 May 2015, Revised 8 July 2015, Accepted 14 July 2015, Available online 17 July 2015

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doi:10.1016/j.bbagrm.2015.07.010

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---

Highlights

•

We generated a human cell line expressing only an analog-sensitive allele, CDK12^as.

•

Our first CRISPR/Cas mutagenesis introduced an unwanted splice site = caution.

•

In the CDK12^as cell line, kinase activity was inhibited by 1-NM-PP-1 in < 15 min.

•

CDK12 inhibition altered CTD phosphorylation, possibly on Ser2 and Ser5.

---

Abstract

The RNA Polymerase II C-terminal domain (CTD) kinase CDK12 has been implicated as a tumor suppressor and regulator of DNA damage response genes. Although much has been learned about CDK12 and its activity, due to the lack of a specific inhibitor and the complications posed by long term RNAi depletion, much is still unknown about the particulars of CDK12 function. Therefore gaining a better understanding of CDK12's roles at the molecular level will be challenging without the development of additional tools. In order to address these issues we have used the CRISPR/Cas gene engineering system to create a mammalian cell line in which the only functional copy of CDK12 is selectively inhibitable by a cell-permeable adenine analog (analog-sensitive CDK12). Inhibition of CDK12 results in a perturbation of the phosphorylation patterns on the CTD and an arrest in cellular proliferation. This cell line should serve as a powerful tool for future studies.¹

[Uhohinc]

What will be interesting to see, is when researchers use the Crisp-cas9 to ........................take a "normal" mouse, and remove its Nfkb gene...........and see what happens.  Or remove the MMD2 gene, which clenches control of p53.

[Uhohinc]

http://grist.org/food/with-lab-grown-meat-can-have-our-animals-and-eat-them-too/

If they can make a bioreactor for "hamburger" or "Steakum" without the entire cow, not long before they can make one's own human melanocytes in a bioreactor, and inject into back into donor human.  Perhaps some "Soylant Green" also.

[Uhohinc]

Basic yeast cells, such as those shown here, can be modified to produce painkilling opiates through the addition of 20-plus genes.

Powerful painkillers can now be made by genetically modified yeast—are illegal drugs next?

Staff Writer

Email Robert

By

Robert F. Service

13 August 2015 2:00 pm

5 Comments

Move over, poppies. In one of the most elaborate feats of synthetic biology to date, a research team has engineered yeast with a medley of plant, bacterial, and rodent genes to turn sugar into thebaine, the key opiate precursor to morphine and other powerful painkilling drugs that have been harvested for thousands of years from poppy plants. The team also showed that with further tweaks, the yeast could make hydrocodone, a widely used painkiller that is now made chemically from thebaine.

“This is a major milestone,” says Jens Nielsen, a synthetic biologist at Chalmers University of Technology in Göteborg, Sweden. The work, he adds, demonstrates synthetic biology’s increasing sophistication at transferring complex metabolic pathways into microbes.

By tweaking the yeast pathways, medicinal chemists may be able to produce more effective, less addictive versions of opiate painkillers. But some biopolicy experts worry that morphinemaking yeast strains could also allow illicit drugmakers to brew heroin as easily as beer enthusiasts home brew today—the drug is a simple chemical conversion from morphine. That concern is one reason the research team, led by Christina Smolke, a synthetic biologist at Stanford University in Palo Alto, California, stopped short of making a yeast strain with the complete morphine pathway; medicinal drug
makers also primarily use thebaine to make new compounds.

Synthetic biologists had previously engineered yeast to produce artemisinin, an antimalarial compound, but that required inserting just a handful of plant genes. To get yeast to make thebaine,

Smolke’s team coaxed the cells to express 21 genes in total, including many added from a diverse set of species (see graphic); making hydrocodone took 23 genes.

Their success, reported online this week in Science, caps a race to install the complex opioid pathway in yeast. Last year, Smolke’s team reported engineering yeast to carry out the tail end of the process, going from thebaine to morphine. In April, Vincent Martin, a microbiologist at Concordia University in Montreal, Canada, and his colleagues said they had created yeast that could go from an earlier intermediate compound called R-reticuline to morphine. A few weeks later, John Dueber, a synthetic biologist at the University of California, Berkeley, and colleagues announced yeast that carries out most of the first half of the pathway, going from glucose to another intermediate compound, S-reticuline. Finally, two groups reported in late June that they had identified the long-sought enzyme needed to carry out the chemical transformation in the middle, S-reticuline to R-reticuline.

A. Cuadra/Science

Even so, many predicted it would take years to put all the pieces together. As it turns out, back in May, Smolke and her colleagues had already largely finished the task. “It shows this field is really moving fast,” says Kenneth Oye, a biotechnology policy expert at the Massachusetts Institute of Technology in Cambridge.

The most important challenge, Smolke says, was increasing the efficiency of each step so losses wouldn’t build up. In one step, for example, a plant enzyme called SalSyn was doing a poor job of converting R-reticuline to another compound called salutaridine. Eventually, Smolke’s team discovered that the yeast made the enzyme incorrectly, attaching the wrong sugars to it. The researchers fixed the problem by reengineering the inserted plant gene.

Smolke plans to go on tinkering. The microbes need to increase output of thebaine by a factor of 100,000 for drug companies to be interested in using them to make medicines. That won’t be easy. But Martin notes that researchers boosted the output of the artemisinin-making yeast by a similar amount. “It will happen,” he says. “The only question is how fast.” Smolke recently formed a company called Antheia, based in Palo Alto, that aims to push that pace.

To keep up with the yeast engineers, Oye says policy experts need to develop rules to limit the risk of unintended uses of engineered microbes. In the case of opiatemaking yeast, such rules might forbid developing strains to produce illicit drugs, such as heroin, and require scientists to build in genes that prevent the microbes from living outside of a controlled laboratory environment.

Not everyone is worried about home-brewed opiates. Andrew Ellington, a synthetic biologist at the University of Texas, Austin, calls such fears “overblown.” The idea that producing vanishingly small quantities of opiates through fermentation is somehow going dwarf the problem of illegal drugs made from poppies is “laughable,” he says. But 
Martin disagrees. “Poppy fields are not readily available to someone in Chicago, whereas yeast can be made available 
to anyone.”

Posted in Biology, Chemistry

Science| DOI: 10.1126/science.aad1613

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[Uhohinc]

http://newsdaily.com/2015/09/u-s-china-uk-experts-to-tackle-vexed-issue-of-gene-editing/

And the Chinese are already tinkering with human embryos thru Crispr cas 9

[Uhohinc]

http://news.discovery.com/space/hawking-earth-is-likely-doomed-humanity-neednt-be-160119.htm?utm_source=zergnet.com&utm_medium=referral&utm_campaign=zergnet_862764
Steven Hawking speaks of a "genetically engineered supervirus"  My guess is the USA, Russia, China, GB, are already working on selective viruses to kill selective types of people/military (maybe Koreans whom like expensive liquor, paranoid only) to have ready and there is no chance they will eventually not be used within the timeline Hawking states. Billions of dollars less to create, and billions of times more deadly than a nuclear fision weapon.

Perhaps with sales report in the next quarterly, we will get time to spend some of the money before Hawkings predictions.

[Michael Street]

That article is published about June of 2015, and every thing in it is true. All the facts are chronological, but it is old. The author may have written that a year or two earlier, than it finally got submitted. I do not know, but it is as if he wrote that before the latest break thrus.

Read this http://www.wired.com/2015/07/crispr-dna-editing-2/ they already are using on a melanoma drug in it near the bottom.

On Tue, Mar 1, 2016 at 10:20 PM, Uhohinc <michaelba...@gmail.com> wrote:

http://news.discovery.com/space/hawking-earth-is-likely-doomed-humanity-neednt-be-160119.htm?utm_source=zergnet.com&utm_medium=referral&utm_campaign=zergnet_862764
Steven Hawking speaks of a "genetically engineered supervirus"  My guess is the USA, Russia, China, GB, are already working on selective viruses to kill selective types of people/military (maybe Koreans whom like expensive liquor, paranoid only) to have ready and there is no chance they will eventually not be used within the timeline Hawking states. Billions of dollars less to create, and billions of times more deadly than a nuclear fusion weapon.

[Uhohinc]

Near the bottom of the above, and here link, is what will be just one of the very helpful aspects of being able to use the ancient ability in Crispr-Cas9 as found in snipping out dna infected in the bacteria dna and then as well in humans, is that mentioned in the wingless gene in spiders, and what will happen when they remove just this one gene.   It will divulge what this gene was in the spider genome for several hundred million years. (FYI spiders or arachnyds do not fly) As in an entire lineage of mice can be created that have just a Braca2 gene removed, and then drugs to test just on these mice.

Interesting that not only are the researchers in this area excited about all the incredible potential, they are worried about abuses. And the simplicity and low cost of Crisp-Cas9

http://www.wired.com/2015/07/crispr-dna-editing-2/

[Uhohinc]

Health

Mar 24 2016, 8:08 pm ET

Synthetic Stripped-Down Bacterium Could Shed Light on Life's Mysteries

by Maggie Fox

&amp;amp;lt;img class="img-responsive img_main-art" src="http://media3.s-nbcnews.com/j/newscms/2016_12/1471986/160324-stripped-down-life-form-01_fb030256c2a8bc5f22c90d6402903e51.nbcnews-fp-1240-520.jpg" alt="Image: J. Craig Venter Institute scientists have created a stripped-down life form" title="Image: J. Craig Venter Institute scientists have created a stripped-down life form" itemprop="image"/&amp;amp;gt;

Electron micrographs of clusters of JCVI-Syn 3.0 cells magnified about 15,000 times. This is the world's first minimal bacterial cell. Its synthetic genome contains only 473 genes. University of California, San Diego

                  

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Scientists have created a stripped-down life form, a bacterium with a minimal number of genes needed to keep it going.

They hope to use it as a platform to create designer life forms, and say it's already taught them some important, and humbling, lessons about the essence of life.

The little bacterium has 473 genes. And the team at the J. Craig Venter Institute in California admit they don't know what a third of the actually do. They just know the microbe dies without them.

"We don't know the key elements of about a third of life," said Venter, who founded the institute.

&amp;amp;lt;img class="img-responsive img_inline" src="http://media2.s-nbcnews.com/j/newscms/2016_12/1471976/160324-stripped-down-life-form-03_fb030256c2a8bc5f22c90d6402903e51.nbcnews-fp-360-360.jpg" alt="Image: J. Craig Venter Institute scientists have created a stripped-down life form" title="Image: J. Craig Venter Institute scientists have created a stripped-down life form" itemprop="image"/&amp;amp;gt;

Electron micrographs of clusters of JCVI-Syn 3.0 cells magnified about 15,000 times. This is the world’s first minimal bacterial cell. Its synthetic genome contains only 473 genes. University of California, San Diego

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But they are close, Venter's team reported in the journal Science.

"It's the smallest genome thus far known on planet Earth of a self-replicating organism," Venter told NBC News.

Venter, who helped lead the effort to sequence the full human genome, has been trying for two decades to create synthetic life. His ultimate goal? A system for custom-making life forms on a computer.

"We don't know the key elements of about a third of life."

"In theory we should be able to add genes to this and evolve it to other species," Venter said.

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But first, researchers have to understand what makes life. To do that, Venter's team started with one of the simplest bacteria known, one called Mycoplasma. Viruses only have a handful of genes - influenza has eight - but they cannot live on their own. They have to hijack a cell. Bacteria have many more genes, are self-contained and can reproduce on their own.

The team selectively took out genes to see what would happen. It's taken a lot longer and was a lot harder than anticipated.

Related: Artificial DNA Controls Life

"I think one thing I've learned from this is that the whole idea of a minimal genome is not quite as clear-cut as it seemed initially," Clyde Hutchison, who led the research, told reporters on a conference call.

They found many genes that didn't seem essential at first, but they learned quickly were part of a pair. One could be taken out, but not both.

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"The idea initially was the minimal genome would have only essential genes, and if you knocked out any gene, it would be dead," Hutchison said. "But the finding of all these quasi-essential genes changes our perspective on this. That if you want a cell that you know grows in a time scale that you can observe, you have to have these quasi-essential genes."

&amp;amp;lt;img class="img-responsive img_inline" src="http://media3.s-nbcnews.com/j/newscms/2016_12/1471981/160324-stripped-down-life-form-02_fb030256c2a8bc5f22c90d6402903e51.nbcnews-fp-360-360.jpg" alt="Image: J. Craig Venter Institute scientists have created a stripped-down life form" title="Image: J. Craig Venter Institute scientists have created a stripped-down life form" itemprop="image"/&amp;amp;gt;

Electron micrographs of clusters of JCVI-Syn 3.0 cells magnified about 15,000 times. This is the world’s first minimal bacterial cell. Its synthetic genome contains only 473 genes. University of California, San Diego

And now they'll have to study the other mystery genes, too.

"It actually taught us that we need to be a lot more humble about basic knowledge in biology," Venter said.

Scientists have been humbled before. They once thought that large stretches of the human genome that did not code for actual genes was "junk DNA". It's now clear that junk is essential for making everything work.

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Other experts praised the work.

"I think biofuels are out of the picture now as the price of oil bottoms out."

It "represents a remarkable landmark in terms of the ability to deconstruct and understand complex biological systems," said Samuel Deutsch of the Department of Energy's Joint Genome Institute.

"It is a very profound result," added Adam Arkin, director, of the Berkeley Synthetic Biology Institute at the University of California, Berkeley.

Related: Genome Pioneer Warns About Biohackers

Venter has some clear goals in mind and his commercial operation, the Synthetic Genomics Institute, was set up to make products using the stripped-down organisms.

"We think the cell would be a great cell to add components to for manufacturing drugs, and complex chemicals," Venter said.

Venter had once hoped to manipulate algae or bacteria to pump out cheap biofuels and had a contract with Exxon to do so. They've since gone back to the drawing board on that one.

"I think biofuels are out of the picture now as the price of oil bottoms out," Venter said. " I think they look a lot more attractive at $200 a barrel than they do at $20."

Maggie Fox

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[Uhohinc]

https://www.sciencenews.org/blog/science-ticker/gene-edited-mushroom-doesnt-need-regulation-usda-says?tgt=more
USDA declines to regulate Crisp9 technology when used to edit out genes from mushroom for marketing of brown free mushrooms.

[Uhohinc]

Risk posed by 'garage geneticists': Fears amateur scientists using DNA editing technology may cause harm

The Nuffield Council on Bioethics warns of risks posed by 'biohackers'
A new report says cheap gene editing kits can be bought online for £100
These can be used to alter the properties of living bacteria or yeast
Biohackers could create something harmful deliberately or accidentally
By Richard Gray for MailOnline

Published: 12:04 EST, 30 September 2016 | Updated: 12:04 EST, 30 September 2016

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Amateur scientists toying with powerful gene editing technology in their garages could pose a future risk to human health and the environment, a new report warns.

It highlights a growing trend for so-called 'biohackers' who use new technology to modify the DNA in living organisms outside of traditional research institutions.

While most of these groups of amateur researchers are attempting to solve problems like creating new flavours of beer, some could do more harm.


A new report has warned that powerful gene editing technology (stock picture) could pose a risk in the future as more people get their hands on cheap kits that allow them to alter the properties of living organisms

The report by the Nuffield Council on Bioethics points to £100 kits that can effectively 'cut and paste' DNA in yeast or bacteria to alter their properties.

HOW DOES CRISPR WORK?

CRISPR-Cas9 technology precisely changes target parts of genetic code.

Unlike other gene-silencing tools, the CRISPR-Cas9 system targets the genome's source material and permanently turns off genes at the DNA level.

The DNA cut – known as a double strand break – closely mimics the kinds of mutations that occur naturally, for instance after chronic sun exposure.

But unlike UV rays that can result in genetic alterations, the CRISPR-Cas9 system causes a mutation at a precise location in the genome.

When cellular machinery repairs the DNA break, it removes a small snip of DNA. In this way, researchers can precisely turn off specific genes in the genome.

This technology, known as CRISPR-Cas9, can be used without needing a high level of scientific knowledge, unlike other gene editing techniques, which need expensive equipment and specialist training.

The Nuffield Council on Bioethics said the 'comparatively low cost, ease of use and availability' of these new gene editing kits, which can be purchased online, meant they were accessible to unregulated users.

This raises the prospect that scientists could either deliberately or accidentally create an organism that could be harmful if released into the environment.

It added: 'These may include DIY 'garage' scientists, school and undergraduate students, and others with an interest in biological research and the possibilities - whether potentially beneficial or harmful - raised by genome editing.'

The report comes just weeks after a leading molecular biologist at Oxford University warned that the technology could be used to develop new types of biological weapons.

Indeed the FBI is so concerned about the activities of biohackers it has set up a special branch within its Weapons of mass Destruction Directorate to engage with them.

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There is a flourishing biohacking community in many parts of the world with amateur groups getting together to develop home-made approaches to studying biology.

FBI WARNINGS OVER GENE EDITING TECHNOLOGY

The FBI has concerns about how this might be used and is attempting to engage with scientists working in this area to help establish ways of tackling potential risks.

Supervisory Special Agent Edward You from the FBI's Weapons of Mass Destruction Directorate said last year: 'Synthetic biology poses some incredible benefits, but there are some risks involved too.

'Now is the time to be able to identify the vulnerabilities and then start setting up mitigation measures.'

In 2010, Mr You warned that as technologies for editing genomes and conducting biological research become less expensive, the risks involved will increase.

He said: 'You're having the barrier of entry to do something mischievous, or actually outright nefarious, getting lower, so individuals or groups will be able to conduct potential harm more so than before.'

For example, one group in London want to fiddle with yeast to alter the flavour of beer, which apparently one group based in London is already attempting.

Biohackers in Japan hope to reintroduce a gene into blue carnations sold in Japan to revert them back to their natural white state.

Another group is hoping to use CRISPR technology to re-engineer yeast to produce a protein found in milk called casein to create a new type of vegan cheese.

The Nuffield report stated CRISPR-Cas9 was even being used by school pupils in a synthetic biology contest called the International Genetically Engineered Machine competition.

Hugh Whittall, director of the Nuffield Council for Bioethics, said: 'There is no evidence that we've seen that there are people with things going on in their garages, but ... this is one of the things that we need to be aware of, be conscious of the possibility.

'It goes back to this question of whether the control mechanism in terms of the supply of the kits and materials is adequate.'

CRISPR-Cas9 was introduced in 2012 and is rapidly transforming biological research.

The system uses certain proteins that allow DNA to be cut and edited at precise, targeted locations.

Human reproduction and livestock farming were identified as two key areas of concern by the Nuffield Council. Both will be the subject of further inquiries by dedicated working parties.

Nuffield Council member Karen Yeung, Professor of Law and director of the Centre for Technology, Ethics and Law at King's College London, said: 'One of the features of this technology is it makes it more accessible to a broader range of users.

'This however has the knock on effect that it may be more difficult to keep a watching brief and monitor effectively what's actually being done in these areas.

The CRISPR gene editing technology has heralded a new era in the ability to edit and modify the genes of living organisms. Some biohacker groups have adopted the technology (CRISPR-CAS9 complex illustrated) to modify yeast and plants
The CRISPR gene editing technology has heralded a new era in the ability to edit and modify the genes of living organisms. Some biohacker groups have adopted the technology (CRISPR-CAS9 complex illustrated) to modify yeast and plants

'We identify that as a potential area of concern.'



In the field of human reproduction, gene editing has the potential to eliminate inherited diseases such as cystic fibrosis.

There are more than 4,000 known single gene conditions that are thought to affect around one per cent of births worldwide.

But producing babies from embryos whose inherited DNA has been altered is illegal in the UK and still highly controversial.

Critics point to the dangers of irreversible changes being passed onto future generations and the possible creation of 'designer babies'.

Prof Yeung, who will chair the Nuffield Council working party on reproductive applications, which meets for the first time next week, said: 'It is only right that we acknowledge where this new science may lead and explore the possible paths ahead.'

CRISPR-Cas9 gene editing can be used to edit the DNA in bacteria like Escherichia coli (pictured). While normally harmless in the cut, these bacteria can also cause food poisoning
CRISPR-Cas9 gene editing can be used to edit the DNA in bacteria like Escherichia coli (pictured). While normally harmless in the cut, these bacteria can also cause food poisoning

Genome editing in farm animals has already been proposed for pigs, sheep, cattle and chickens, raising questions of food safety and animal welfare.

Animals whose genes have been edited may fall into a grey legal area because it is not clear that their meat, eggs or milk would be classified as genetically modified food.


Potential applications of the technology included pigs protected against swine flu, chickens that only produced female offspring for egg production, and hornless cattle that could safely be kept in confined spaces.

Dr Andy Greenfield, from Oxford University, who chaired the review working group, dismissed suggestions that farmers might secretly make use of gene editing technology.

He joked: 'A kind of evil Old Macdonald? There are entry barriers. It's not that it's so easy to do that you can just have a shed at the end of the field.

'It would be difficult to do this secretly. It's not so quick and easy that it could be happening across Suffolk.'

Dr Greenfield was most concerned about 'frivolous' use of CRSPR-Cas9 gene editing.

'We don't really want to encourage frivolous cosmetic uses of a powerful technology when we have real needs right now,' he said.



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Read more: http://www.dailymail.co.uk/sciencetech/article-3815998/Risk-posed-garage-geneticists-Fears-amateur-scientists-using-DNA-editing-technology-cause-harm.html#ixzz4MHetHqBG
Follow us: @MailOnline on Twitter | DailyMail on Facebook

[Uhohinc]

CRISPR Corrects Sickle Cell-Causing Gene in Human Cells
Once implanted in mice, the edited stem cells produced normal hemoglobin.

By Kerry Grens | October 13, 2016
5285

WIKIMEDIA, DIANA GRIB
Scientists have used the CRISPR-Cas9 gene-editing technique to rewrite the genetic mutation in blood cells that causes sickle cell disease. Once these treated hematopoietic progenitors, which had been harvested from patients, were given to mice, the cells began to produce healthy hemoglobin.

“What we have right now, if we can scale it up and make sure it works well, is already enough to form the basis of a clinical trial to cure sickle cell disease with gene editing,” study coauthor Mark DeWitt, a postdoctoral fellow at the University of California, Berkeley, told The Los Angeles Times. His team published its results yesterday (October 12) in Science Translational Medicine.

As STAT News pointed out, the technique is not 100 percent efficient. Only a fraction of the treated cells successfully ended up with the right edits; and only 2 percent to 6 percent of the corrected cells retained the edits after 16 weeks once administered to the mice. “A few percent might seem low, said Jacob Corn, scientific director of the [UC Berkeley] Innovative Genomics Initiative and a co-leader of the study, but studies suggest that having just 2 percent to 5 percent of healthy red blood cells could be enough to cure sickle cell,” STAT reported.

Stuart Orkin, a blood stem cell biologist at Harvard Medical School who was not involved in the work, told Chemical & Engineering News that CRISPR is just one route to repair the gene that causes sickle cell anemia. “There is heightened interest given the hoopla around CRISPR/Cas9 but there are other ways to edit,” Orkin said. “Ultimately, the approach that achieves excellent patient outcomes without side-effects is what is desired and there may be several different ways to achieve this end.”

[Uhohinc]

I think this above has tremendous implications.

[Uhohinc]

https://www.youtube.com/watch?v=2pp17E4E-O8&autoplay=1 The video titled "Designer Babies" gives a simple presentation of how Crisper Cas-9 is already looking very promising. I think it is missing the negative possability and abuse. Just like in the Michael Crichton Jurassic Park books and movies and all the way back to his 1970s Andromeda Strain was art ahead of reality. I think it would be a dereliction if every modern military is not contemplating its use and counter measures now.
This is also why I do not think it will be decades as the video states. I think the Chinese will accelerate the technology and bring it on with astounding progress. Being the technology is easier for now to change the one cell embryonic zygote, i think the super humans will start in a year or two. And all genetic disease such as the porphyrias will be gone in these newborns. I wonder what attributes Chinese parents will choose. Even the gene that determines how the bridge of the nose is distanced is known. The blue eye gene is easy to do with one gene. And cosmetically coveted lighter skin tone is being bleached can be chosen.
Intelligence is for now too many genes and transciptioning and not just one. So awhile to make a super musician or coordinating muscle memory athlete Etc. And dangerous to change several genes that are not definitely understood beyond simple off on. The genetic cause of say autism or child molesting propensity is not known. Or it may be not genetic but a pathway signaling or unknown other causes.
It may lag in time and be more difficulty to figure out and to make changes to a living persons genome, but probably work. Though there may be a difference between "us" and the new class of super humans whom will be super healthy and tall and thin and smart and who will not die but for accidents or killing. No more races of humans just hybrids.
We now alive may be treated as today the developed world ignores or indifference to several equatorial African country's which have half their populations below 70 percentile IQ of an " normal " 100 IQ average of most Northern Hemishpere orgined genetic sub ethnicity profiling. Maybe another century till as many think that humans will become pets of our to be created computer android masters. Or as some think we will download are entire reality into a super digital computer that humans are going to develop........or that some being already did to us and that's where our reality really is.

[Uhohinc]

I just thought how the future United Nations can pass a law that all designer humans must be one tenth the size of today's human. This would make natural earth resources and energy go a thousand times more. No more traffic, more beach front housing, smaller cars and planes. Then they would not need to genetically modify cows or crops.

[Uhohinc]

Simulated reality is the hypothesis that reality could be simulated—for example by computer simulation—to a degree indistinguishable from "true" reality. It could contain conscious minds which may or may not be fully aware that they are living inside a simulation.
Simulated reality - Wikipedia, the free encyclopedia
Wikipedia › wiki › Simulated_reality
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Simulated reality - Wikipedia, the free encyclopedia
Wikipedia › wiki › Simulated_reality
Simulated reality is the hypothesis that reality could be simulated—for example by computer simulation—to a degree indistinguishable from "true" reality. It could contain conscious minds which may or may not be fully aware that they are living inside a simulation.
‎Types of simulation · ‎Arguments · ‎In fiction · ‎See also
Simulation hypothesis - Wikipedia
Wikipedia › wiki › Simulation_hypothesis
The simulation hypothesis contends that reality is in fact a simulation (most likely a computer simulation), of which we, the simulants, are totally unaware. Some versions rely on the development of simulated reality, a fictional technology.
Are We Living in a Computer Simulation? - The Nature of Reality — The Nature of Reality | PBS
PBS › nova › blogs › physics › 2015/07
Jul 8, 2015 - if we live in a simulated reality we should expect occasional sudden glitches, small drifts in the supposed constants and laws of Nature over time, and a dawning realization that the flaws of Nature are as ...
Elon Musk Says There's a 'One in Billions' Chance Reality Is Not a Simulation | Motherboard - Vice
Vice › motherboard › read › elon-musk-...
Jun 2, 2016 - Elon Musk says he firmly believes reality is a simulation created by a superintelligence.
Are We Living in a Computer Simulation? - Scientific American
Scientific American › article › are-we-livi...
Apr 7, 2016 - The idea that the universe is a simulation sounds more like the plot of “The Matrix,” but it is also a legitimate scientific hypothesis. .... Reality Check: You Are Not

[Uhohinc]

http://motherboard.vice.com/read/elon-musk-simulated-universe-hypothesis

[Uhohinc]

Scientists restore hearing in deaf mice using advanced gene therapy
Published time: 7 Feb, 2017 01:45
Get short URL
Scientists restore hearing in deaf mice using advanced gene therapy
© Vin Catania / AFP
91
Scientists have restored hearing in deaf mice down to a whisper using an improved gene therapy in what is being described as a “landmark” study with “unprecedented” results.
A research team from the Boston Children’s Hospital initially conducted a study with Harvard Medical School in 2015 that restored rudimentary hearing in genetically deaf mice using gene therapy.

In this new study, however, the Boston team managed to restore hearing in deaf mice down to 25 decibels - or the equivalent of a whisper - using an advanced gene therapy developed at Massachusetts Eye and Ear.

Read more
© imagebroker / Alfred SchauhuberMind control lasers turn mice into killer rodents with the flip of a switch
The team said they sought to advance gene therapy to treat genetic deafness as there is currently no biological treatments for hearing loss.

“We focused on Usher syndrome, a devastating genetic disorder that causes blindness, balance disorders and profound deafness,”read the study, published in Nature Biotechnology Monday.

Using a synthetic adeno-associated viral vector, the team transduced 80-90 percent of sensory hair cells, resulting in a “recovery of gene and protein expression, restoration of sensory cell function, rescue of complex auditory function and recovery of hearing and balance behavior to near wild-type levels.”

The end result of the study saw an “unprecedented recovery of inner ear function” that suggests the biological therapies may be suitable to treat deafness in humans with genetic inner ear disorders.

"With more than 100 genes already known to cause deafness in humans, there are many patients who may eventually benefit from this technology,"said Dr. Konstantina Stankovic, a senior investigator in the first study with Harvard Medical School.

READ MORE: Rat-mouse interspecies transplant brings hope human organs could be grown in animals

The next step toward treating human patients is to test the gene therapy in larger animals.

"This is a landmark study,"says Dr. Jeffrey R. Holt, director of otolaryngology research at Boston Children's Hospital, and co-author on the paper.

"Here we show, for the first time, that by delivering the correct gene sequence to a large number of sensory cells in the ear, we can restore both hearing and balance to near-normal levels."

[Uhohinc]

https://m.youtube.com/watch?v=jAhjPd4uNFY

Video simplificated understanding of Crspr cas9

[Uhohinc]

SALK INSTITUTE

Magazine issue: Vol. 191 No. 3, February 18, 2017, p. 6
Chimeras

Growing human organs in other animals is a small step closer to reality.

Injecting human stem cells into pig and cattle embryos created embryos that incorporate a small number of human cells, scientists report January 26 in Cell. The ultimate goal of the controversial research is to use hybrid, or chimeric, animals to produce human organs for transplant.

Farm animals incubating human organs won’t appear anytime soon, says Juan Carlos Izpisua Belmonte, a stem cell biologist at the Salk Institute for Biological Studies in La Jolla, Calif. “I feel we’re still far away from that,” says Belmonte, who led the work. It has taken his group four years “just to deliver a message that, yes, human cells can integrate into a pig.”

While human-animal chimera work is still in its infancy (and faces ethical and funding hurdles, see sidebar), hybrids of rats and mice are already hinting that growing an organ from one species in another is a viable strategy for curing some diseases. Researchers report January 25 in Nature that they grew mouse pancreases in rats. Mouse insulin-producing cells were extracted from the rat-grown organs and transplanted into diabetic mice, curing their diabetes. Transplanted cells kept the mice’s blood sugar normal for more than a year even though the mice were not given immune-suppressing drugs to prevent rejection after the first five days following the transplant. That finding raises the hope that animal-grown organs tailored to individual patients could be transplanted without fear of rejection.


TOUCH OF HUMAN Human cells (green) that were integrated into pig embryos were able to form cells (red) that give rise to the lining of the gut and other tissues. Shown is tissue from a 4-week-old pig embryo. DNA in both human and pig cells is shown in blue.
SALK INSTITUTE
Chimeric animals get their name from a fire-breathing monster in Greek mythology that had the head of a lion, a goat’s body and a snake’s tail. Researchers create patchwork animals by injecting early embryos of one species with stem cells from another species. In 2010, Hiromitsu Nakauchi, a stem cell biologist at Stanford University School of Medicine, and colleagues reported making a mouse that grew a rat pancreas. The researchers were surprised to find that the organ grew to the size of mouse pancreas, instead of creating a much larger rat-sized organ.

The mouse-grown pancreas wasn’t big enough to transplant back into a rat. So for the new study, Nakauchi and colleagues reversed the experiment, growing a mouse pancreas in a rat. The researchers used a genetic trick to ensure that the pancreas was composed mostly of mouse cells, breeding rats that cannot make a pancreas because of defects in the Pdx-1 gene. Normally rats without a pancreas die shortly after birth. But animals that got mouse cells were able to grow a functioning pancreas.

In the study reported in Cell, Belmonte and colleagues also worked with rodents, creating mice growing rat organs. Using the gene-editing tool CRISPR/Cas9, the researchers disabled Pdx-1 and genes involved in heart and eye development so that mice couldn’t grow functioning versions of those organs. Rat stem cells injected into mouse embryos filled in, growing functional organs, including one the researchers didn’t expect. Rats don’t have gallbladders, but rat stem cells introduced into mice embryos are able to form gallbladders, the researchers reported.

This finding illustrates that the host environment has significant control over what happens to stem cells, says Daniel Garry, a transplant cardiologist and stem cell biologist at the University of Minnesota in Minneapolis. “That’s pretty cool biology.” The result also indicates that it may not be possible to re-create in lab dishes the mechanical forces, chemical signals and other conditions an organ needs to develop properly.

In the case of the human-pig chimeras, researchers weren’t sure how well the human cells would fare, Garry says. For all scientists knew, human stem cells might take over pig embryos. “That would be very much unwanted,” Garry says.


But the Salk researchers and colleagues found that human cells don’t incorporate efficiently into pig embryos. Of 2,075 pig embryos injected with human stem cells and transferred to sows’ uteruses, only 186 grew for 21 to 28 days — as long as the researchers allowed development to continue. Of those, 67 contained human cells. Most of the chimeric embryos were underdeveloped, indicating that human cells may interfere with normal pig development. Early cattle embryos appeared to accept human cells more easily, but cows are more difficult to work with than pigs.

More work is needed to improve growth of human cells in pig embryos. The Salk researchers hope to use CRISPR/Cas9 to engineer pigs to lack certain organs just as they did with rodents, says Jun Wu, a stem cell biologist on Belmonte’s team. Human cells able to supply the missing organs might have a growth advantage and survive better.

Research Hurdles
Some people are not excited about the prospect of growing human organs in animals. “People view chimeras as monsters,” says Stanford stem cell biologist Hiromitsu Nakauchi, who is attempting to make pigs and sheep that carry human organs intended for transplant.

Part of the discomfort many people feel stems from concerns that the chimeras might be “too human,” giving pigs human brains or raising the possibility that breeding a chimeric animal could produce a human baby, says Robert Streiffer, a bioethicist at the University of Wisconsin‒Madison.

In some countries, creating human-animal chimeras is banned. Researchers in the United States are allowed to conduct human-animal chimera work using private or state funding, but not with federal money.

The U.S. National Institutes of Health currently has a moratorium on funding such research. In August, NIH announced intentions to lift the funding ban pending finalization of policy governing such research, but there is no final regulation yet. The proposed rules would expand prohibitions on introducing human stem cells into early nonhuman primate embryos and strengthen bans on breeding any animal that might produce human eggs or sperm, says Carrie Wolinetz, associate director of science policy for NIH. A new oversight committee would also be created to address issues of ethics and animal welfare raised by such research, Wolinetz says. “We’re trying to allow the research to move forward responsibly,” she says.

Bioethicist Françoise Baylis contends that there is no ethical way to continue chimera work because it is based on a faulty assumption that human life is more valuable than that of nonhuman beings. “The hope that one can ‘forever’ avoid the tough ethical questions by simply ensuring that the nonhuman animals are not ‘substantively humanized’ is flawed (short-sighted),” Baylis, of Dalhousie University in Halifax, Canada, wrote in an e-mail.

[Uhohinc]

http://www.dailymail.co.uk/sciencetech/article-4211206/Biohackers-Germany-face-jail-time.html

$349 for a bio hacking kit. I can not see how this can be policed from mischievous individuals, or a intent on terror killing group.

[Uhohinc]

https://medicalxpress.com/news/2016-11-chinese-group-crispr-cells-human.html#nRlv
The Chinese researchers alter the PD-1 gene (cell death involved) from a human and re-inject.

[Uhohinc]

http://gizmodo.com/heres-why-todays-decision-over-who-invented-crispr-matt-1792402072

Everything can be entirely lost in the patents

[Uhohinc]

http://metro.co.uk/2017/02/19/bill-gates-thinks-theres-going-to-be-a-global-pandemic-that-will-kill-millions-6458780/

I do not see how Gates thinks it will only kill 30 million. He should have seen this two years ago and press released. This makes any anx about climate change trivial. That N Korean dictator killing his half brother with an unknown chemical in his face was probably tested on a lot of now deceased involunteers.

It will take awhile to selectively kill say a religious propensity, or ethnicity or nationality or sexuality or just ranking officers is difficult for now, it's easier to kill everyone. The Apollo moon landings bringing back a microbe fear had the astronauts locked up airtight for several days as caution.
3,000 years of conventional War has been obsolete where the one with the bigger army won. Unless they go back to killing the entire population as I think happened to several millions of Uhrs by the Persians. In the Gates video is another reference to the present day exterminating of Yadzi.

I see engineered microbes or bacteria at first not killing humans, as it will kill the human designer also. I think they will use it indirectly to maybe kill all the pollinating bees. Or there are about 3 billion pigs per year in world they could bring back swarms and plague to effect food supply or economics regionally.

Those 9/11 16 hijackers actions on just 3 planes caused now several trillion dollars directly in responses.
I think most civil rights and any privacy and personal interrogation with about a hundred times the present law enforcers with no search warrants will dwarf the "patriot act"
. With the ease of evil genetic editing, today's teenage internet hacker will be tomorrow's mischievous bio hackers. I can not see how fascism and police state will not be the eventuality worldwide. The nuclear genie is already no compare to the gene genie.

Might be a good idea to not live in a major Western city in a few years.

[Uhohinc]

https://www.sciencenews.org/article/gene-editing-human-embryos-yields-early-results

Chinese claim early success in editing out the bad genes in a human (embryo). My guess is they used a healthy 23 chromosome human embryos, and just keeping the West from getting in an ethics headache. To really test crispr cas9 is to see what complications arise I would think they would let it go to birth.

[nosuchth...@gmail.com]

We won't be needing any medications soon will we. 10 years? 15 years max!

[Uhohinc]

I just went back to see how long ago the Chinese announced their first Crspr cas9 in a human. I posted that in July 2016 where the Chinese publicsized the gene edit. And today is if not 9 months it will be, but we do not know the for sure start date. And coincidentally not it takes nine months fro the first human cell that is a fertilized female egg. I assume the Chinese started as the very first cell before it divides collectively trillions of times in nine months to be a full term baby.

Of course it would only make sense to change the first cells DNA. And for them to deem it a success I think the Chinese put that zygote fertilized human egg back into a or some females womb to let it develope.

I think the Chinese infer the study is ongoing so I think they let the baby birth or it will very soon. And the Chinese are not bound by all the ethics study's and politicizing the Americans and French and British keep meeting and setting down rules.

I do not think it will take ten years because of the Chinese are going to make the private investors move quicker. Recall how the Chinese only slow one child and will fine any one whom has a second baby and this resulted in tens of millions of female fetus abortions. It is still not known at zygote if the baby will be a boy or girl.

My guess is the first gmo baby was just born in China. With about 7,000 human identified disorders, and EPP is just one. They will want to identify the major ones first. But how commercially tempting will it be to change one gene so your child most assuredly has green or blue eyes.

There are several genes attributed to making one very religious. Whom would not want a baby with a 140 intelligence quotient.

For us already alive and made of two trillion cells already it will not be as easy as in a new one cell designer human for now.
The Chinese will work first on perfect gmo farm animals. I think I recall the Chinese slaughter over two billion hogs a year.

EPP will be gone and a footnote I would think in newborns that most all here do not comprehend how polio was so involving in everyone's life. And tuberculosis, consumption, diphtheria, small pox etc....but if a pregnancy is not intended or planned I do not see many using crispr cas9. I think to difficult to pull the fetus out after to many divisions in a few days. Unless they perfect the edit or automate it. They will.

We already alive will have to wait for personalized stem cells grown in the marrow put in a lab and multiplied in the millions and intermittently re injected in to needed organs, but that just happened at least in bone marrow stem cells.

Here EPP looks like a very difficult for now in adult EPP patients. EPP is in blood cells erythrocytes the red blood cells. And they renew from stem cells coming from marrow
With crisprcas9 and looking at how I think they would first attempt it they will have to kill the entire marrow and do a marrow transplant of a very good and healthy donor or need to introduce I think a million gene edited out the EPP causing gene and put them back in the marrow and then monitor the immune attacks. Scenesse is much less risky for now.

They have to find a way I'm not aware of to edit stem cells of each individual and then take out the gene isoforms types that cause a certain disorder. I have not read this anywhere but seems like it can be done soon. But stem cell therapy appears great for long lived cells in humans as they can be reinjected intermittently as replacements.
But everyday a human looses tens of millions of blood cells, intestinal wall cells, skin cells. Melanocyte stem cells would be a easier target. As well neurons, and most others. The stem cells in the eyes makes sense.

[Uhohinc]

https://www.sciencenews.org/article/crispr-had-life-it-became-gene-editing-tool?mode=topic&context=87&tgt=more

[Frank1895]

Am Mittwoch, 3. Mai 2017 06:00:45 UTC+2 schrieb Uhohinc:
> https://www.sciencenews.org/article/crispr-had-life-it-became-gene-editing-tool?mode=topic&context=87&tgt=more

Hello Uho,

by the ongoing development of Crispr-Cas9, how do you see the future of Scenesse...?

[Uhohinc]

Crspr Cas9 technology is for now easier suited for changing embryonic stem cells....way befor to many have already committed and populated a new life. Be it a cow or a fruit or a new type of animal it will be easy to start anew and create a developing embryo to animal. One cell divides multiple times and it's offspring divide to make trillions of cells, so it makes a difference to change the very first cell or the closer to it so as to pass on the modification.

Not so easy to repopulate a child or adult human with re-engineered cells with genetic changes as they already have several hundred cell types in the trillions that will keep the same profile of how they make or do not make their proteins relative to their genetic start from birth when 23 pairs of chromosomes were half contributed from sperm and half from egg. It is successful to an extent, but will have to keep an oversight as the technology progresses. No doubt it is going there, just how soon. As with Scenesse, nothing happens fast in biotech or drugs. As always a couple more years.

[Frank1895]

Am Donnerstag, 4. Mai 2017 01:12:17 UTC+2 schrieb Uhohinc:
> Crspr Cas9 technology is for now easier suited for changing embryonic stem cells....way befor to many have already committed and populated a new life. Be it a cow or a fruit or a new type of animal it will be easy to start anew and create a developing embryo to animal. One cell divides multiple times and it's offspring divide to make trillions of cells, so it makes a difference to change the very first cell or the closer to it so as to pass on the modification.
>
>
>
> Not so easy to repopulate a child or adult human with re-engineered cells with genetic changes as they already have several hundred cell types in the trillions that will keep the same profile of how they make or do not make their proteins relative to their genetic start from birth when 23 pairs of chromosomes were half contributed from sperm and half from egg. It is successful to an extent, but will have to keep an oversight as the technology progresses. No doubt it is going there, just how soon. As with Scenesse, nothing happens fast in biotech or drugs. As always a couple more years.

Thank you for the answer, Uho...

[nosuchth...@gmail.com]

WOW!! "A couple more years"

Scenesse is a proven safe and extremely effective drug for this and many other similar conditions. CS9 will one day fix every human fault but we should all be rich by that time. People with EPP can live a normal life now with the most minimal of negative side effects so why would they want to mess with Gene manipulation when their is so much unknown. If FDA ever green light Scenesse it will be the go to product for a decade or more unless some insane scientific breakthrough happens but I see it all going back to efficacy and safety of Scenesse luckily.

[Uhohinc]

http://www.medicalnewstoday.com/articles/317301.php This is another fundamental breakthrough tangent of Crispr Cas9 . We are all accumulating virus through life. Some lead to cancers. I'm not sure how they are doing this, but it will fast forward attract research dollars and focus which will accelerate the crispr cas9 applied science to the patient bedside sooner.

EPP patients problem is in the blood cell erythrocytes lacking an genetic marker for heme production that most everyone else has. Hemophiliac patients will be way before EPP patients, but the procedure will probably be very identical. And I do not think to far off.

I think they will extract an hemophiliac or EPP patients stem cells in the marrow of their thigh bone with a needle. They use Crispr Cas9 to snip out the known DNA string for where the instruction for the heme protein in the case of EPP is and replace it with a gene sequence known to properly produce the correct heme protein.

Just one small gene area change. Not like a complicated genetic disorder of many genes or unknown location of DNA causing such and such of the 6000 plus gene disorder diseases.

So they change a few red blood cells and then grow them in the lab, stem cells specific to that patient. They make hundreds of millions ( note the link I included as to this breakthrough last month) of these corrected red blood cells and every month or two have these red blood cells transfused into the same EPP patient. To note, red blood cells on average only survive in most humans about 3 months. I read this plausible in all the marrow based blood immune cells: basophils, esonophils, macrophages, T cells, monocytes, Astrocytes, (that's all I can think of now) but it's not so easy to extract a persons melanocytes, grow millions more and re inject.......yet.

I see this happening sooner than a elaborate Crispr cas 9 edit that is done from the stem cell source in bone marrow.

I think I read into it that any of

[Uhohinc]

https://www.nature.com/articles/s41598-017-01727-y

Crispr Cas9 used on rabbits to eliminate the tyr gene to determine effects in melaningenisis and albinism. Dramatic pictures.

[nosuchth...@gmail.com]

I'm just going to pretend I didn't see that! Seriously though it would have to be at least 10 years away from human use!?

[ffuc...@gmail.com]

OK, my question again,in other words: Does Scenesse have a future, after CC9 hits the market?

[Uhohinc]

In one word yes, but for how long and what for................... You demand a simple answer to the prediction of future that is yes or no, bottom line with no complicated thought and many unknowns near term let alone longterm, but the real world outside of a teacher like test does not work that way. Not a multiple choice question with only four answers and one correct. To many variables and future buts or ifs. You ask a yes or no answer to a question with millions of multiple choice answers. Then there's timing, if we do not see what's coming to soon or to far off then we spend a decade to long in a stock waiting for the future or miss out by missing what's about to happen.

Yesterday I would never have thought that some research lab would for the foreseeable several years at least do what they did with that Tyr gene to see what it did to that rabbits hair, skin and eyes. EPP and thousands of other diseases are also a one gene disorder in humans. I would think after ten years of look and see research that every pregnant women will go in for a sample of the baby DNA and an assessment of all the genes alleles( that are known to cause the known disorders will be corrected with a deletion and insertion with a established and known allele that does not cause such protein deficiency. In humans worldwide there are over 80 types or alleles of different humans whom have a different version of mcr1 gene.

Humans have about 26,000 genes, but some are not simple. They are transcription factors. Or changing two or say a hundred changes another five hundred, which then change another 3,000 and so on is for now just not comprehensible as it is trillions of possibilities in factoring effect.
But a lot better than now then giving a trial and just not really knowing what's going on but just wait and hope the p values show a outcome that's better than the negative.

I think a Boeing Dreamliner or space shuttle is more complex than a human if just by the factors of numbers and systems connected, made of millions of parts, but which ones can fail or be removed or specs changed and still get it to fly. The EPP gene is just one tiny part of the cardiovascular system and not super integral to that system or inrtfacing with other body systems to system function or entire human to be a threat of failure or death.

[Uhohinc]

https://m.phys.org/news/2016-05-melanin-great-batteries.html
In about 1880, the technology breakthrough high tech and future was with the up and coming Kodak. And for a hundred years it made many rich and could not misstep. Then Fuji brought major competitive products, but even til about into the 2000s Kodak was still a 35 billion dollar market cap. Digital technology was a disruptive technology but it did not sneak up on the investors whom still held on to its stock for so much so long. Most all of that paper billions was only in their minds, it disappeared.

Fracking technology has been disruptive technology to very worried Saudi oil and banking interests. But the technology has been evolving since the 1890s. Note that three years or so ago, the Saudis flooded the market with oil to intentionally drop Brent crude price to with purpose bankrupt the American smaller oil producers whom were eking a profit at higher production. This worked temporarily with wells shut down and drilling drastically cut back, but it culled the weaker oil producers, consolidated the survivors to be stronger, and sped up the technology to get even more oil at lower cost out of what has been unrecoverable oil always left in the ground.

Crispr Cas9 is hugely major disruptive technology in not just pharmaceuticals, but in farming, animal foods, sewage water treatment, health insurance, warfare, energy and on and on. But the changes in technology will be much faster and much less warning then Kodak or the Saudis got.
Technological obsolescence is going to be a fast threat to every stock.

[Uhohinc]

https://en.m.wikipedia.org/wiki/Kodak

[Zero tolerance]

Do you think the good Doctor sees this as a short term threat to Scenesse?
More specifically, with his 3 million+ shares, do you see, or foresee, him making decisions based on this imminent technological advancement?
Obviously he has proceeded meticulously through the regulatory obstacle course thus far, but could such a competitive threat speed anything along, such as a buyout?

[nosuchth...@gmail.com]

Scenesse is incredibly safe and super effective. CC9 would cure the underlying issues of EPP but would do nothing in the way of providing photo protection.
Scenesse provides more benefits than CC9.

[nosuchth...@gmail.com]

I should have said CC9 will not give "superior photo protection" if it were to cure the underlying issues concerning EPP like Scenesse will. For the next 10 years I hope☺️.

[Uhohinc]

Just like they changed the one tyrosine gene in the rabbit and interfered with all melanin production in that rabbit and it's offspring eye pigment, hair color and skin to cause a better tan could be several other one gene changes. The easiest target to cause a quick and easy tan would be to remove the versions of mcr1 gene that are common to Northern European Fitzpatrick scale 1 and 2 or even 3 and reinsert one of the 70 or so other in the population mcr1 genes which cause a tan fast. African Negros have essentially no more melanocytes in their skin than a Scottish red haired type. Most of the variations in mcr1 by far have been in European orgined populations.

There are technical hurdles yet, but the simple breakthrough of copying how bacteria self defend and use Crispr Cas9 to remove viral inserted genes in themselves is a process genetic engineering huge technical breakthrough which had stymied biologists since the 1970s and Dolly the sheep. And the first attempt approved by FDA trial in humans was stopped because it killed the first patients with using a virus natural ability and piggybacking into the human cell nucleus and haphazard insertion.
They spent 40 years trying to perfect that genetic engineering method and Crispr Cas9 was paradigm.

How they snipped out the hiv acquired immune deficiency virus in the humans immune cell cd4 cell I understand ( posted last week) but how they repopulated all these cells in the human and essentially cured the hiv I do not understand. Waiting for more info.
This is how I understand it.
The cd4 cell, which msh is very involved in, is the very immune cell in humans which job is to go around and receive signals and or seek out viruses of any type which have entered a humans cells and or have orchestrated ones cells to make more virus rna and bursting open to release this new copy's of such virus. Cd4 immune cells mostly but the entire immune cells when healthily responding keep such virus in check. Not always as in Ebola virus.

The sneaky thing about hiv is which cell it has evolutioned to hibernate and insert its viral rna strand into, and thus evade the immune system response...........it hides in the very cell which is supposed to find and kill the cell itself.....the cd4 cell.

AIDS patients die from simple infections or cancers that a normal immune response can easily control.

Though I can see it easier to introduce engineered blood and immune cells easier than other longterm body cells like a melanocyte, for now

A melanocyte is like an octopuss with tentacles. These moving arms that come out of a melanocyte are moved as one melanocyte services its melanosomes to its affiliated 30 to 36 adjacent keratinocytes.
I do not think one can just inject more engineered melanocytes into the subdermis and expect them to repopulate. I do not know how the harvesting of melanocytes and replanting in Vitiligo is a uccess.

These people that are going to Mexico, India and China awith claims of stem cells farmed from blood, isolated and concentrated into a bad heart or organs is junk medicine playing on people's hope and fear and money, none have been very successful in overall outcome.

[Uhohinc]

Nothing short term here. Remember we have been hearing of tissues engineering and organ replacement regenerative medicine for over a decade. But The technology vanguards in concert and the FDA political shakeup appear to maybe accelerate many aspects in medicine and regulatory timelining

[Uhohinc]

I think Crispr Cas 9 technology, which has already seen tremendous improvement in just the last year will go much faster than the ' its ten tears away" cliche for three reasons, the first two mostly.

First it transcends to many human industry's, business,s but importantly is not just corporate but country's. The USA has secret undisclosed budgets of about $50 billion in CIA/milatery industrial complex black or skunkworks projects. These defense research projects are what really made many things happen and nurtured technologies since WWII as in the internet, the computer, electronics, microwaves, transistors, satellite offshoots, gps, rockets and planes, nuclear power, and on and on and on.

The national defense of many nations will recognize that each must be first and fast to be on top in this new and very important commercial technology. The ability to now create a new man made strand of engineered DNA that can be introduced into humans like a virus and enter a cell DNA string in the billions in length precisely where wanted is just to much a opportune. A bomb that does not kill but incapacitated maybe.

The second major reason is that the technology really does not have to need 10 more years. It is already here, it works very welll success rate that's getting better, and it is very cheap to do. The aspect of embryonic genetic engineering is probably happening in every university this year. Albeit the technology to alter non embryonic needs some technical leaps.

Ethically or unethically almost every suspected Down's syndrome (and other disorders) pregnancy embryo fetus is aborted in Europe USA. The fertility clinic business is in the billions now, For not even a thousand dollars of equipment any clinic can soon pick gender, eye color, etcc but not all is known of all the genes.

They are going to start mapping now. Rats and mice are not close enough to humans. They will start with monkey embryos and one buy one remove every single gene and see what affect it has after birth. Then combinations of gene removal. They are going to need a couple hundred thousand monkey embryos very soon.
Now how long before a student in a lab inserts some human sperm DNA into mice for fun to see what is born.

The third possible reason the technology may warp speed is this is like 1978 and the personal computer age, yet this time everyone knows it's future is big. And all it takes is one of the hundreds of silicone valley billionaires adverse to ageing and looking to invest ala Elon Musk or Sean Parker or Peter Theil type or that San Diego Craig Ventner that developed artificial life and was fired by NIH for his idea saving the government 30 billions and 20 years in the human genome cataloging with his DNA copying machine.

We will have to keep a eye on how quick the future breakthrus are public or figure out what's kept under by corporations and governments. I would not put it past the Chinese to tell everyone it fails to gain advantages they already have in taking the lead.

[nosuchth...@gmail.com]

Please don't tell the guys over on SS.

[ffuc...@gmail.com]

Hello Uho, thanks for your thoughts and time... We will really have to watch the market even more close...

[Uhohinc]

https://medicalxpress.com/news/2017-05-gene-therapy-vision-loss-proven.html#nRlv This may be one way of introducing altered genes into a cell.

[Uhohinc]

https://www.sciencedaily.com/releases/2017/05/170511083806.htm Islet cells successful transplanted to become pancreatic insulin cells utilizing stomach lumen.

[Uhohinc]

https://www.statnews.com/2017/07/28/cell-build-from-scratch/

Craig vintner ventner. Build a cell Cal tech creating a cell to do things.

[Uhohinc]

http://ipscell.com/2017/08/4-reasons-mitalipov-paper-doesnt-herald-safe-crispr-human-genetic-modification/ Impediments and technical aspect of Crispr

[Uhohinc]

http://www.foxnews.com/science/2017/08/11/mutant-ants-created-with-gene-editing-technology.html

Mutant ants thru gene edits results in abherent behavior

[Uhohinc]

https://www.sciencenews.org/article/gene-editing-creates-virus-free-piglets?tgt=nr Gene edited piglets born without virus

[dan.mur...@gmail.com]

Crispy-Cas9 was used in research to discover why miscarriages happen to 1 in 4 women. They have made a connection between lack of vitamin B3 and insufficient amounts of a particular protein in test done on mice which resulted in miscarriages. Similar to the folate problem but much more dramatic in terms of essentially being able to decrease the number of miscarriages through taking a supplement. Still plenty of research to do I'd imagine but I don't think it's too early to call this a breakthrough. Well done to them.

[Uhohinc]

https://www.nature.com/articles/s41598-017-08636-0?WT.feed_name=subjects_genetics CRispr Cas9 used in China to change sheeps coat color

[Uhohinc]

https://endpts.com/building-up-its-io-ops-crispr-therapeutics-allies-with-marcela-maus-at-mass-general/ This editing of B cells and T cells to better recognize and attack ones cancerous cells is looking simple. There's over 100 types of human cancer. But just with melanoma, there are about five subtypes of this cancer which all of these can utilize several different pathways-------always making attempts to survive and proliferate by throwing up several different proteins s to block the immune cells from trying to identify or un-hide them. And even then they adapt and utilize other proteins to stay immortal.

The US "war on cancer " started in early 1970s appears to be at just one therapeutic here with Crspr Cas9 that it is getting very likely to end cancer in animal clinical trials within a one year.

Time for the actuarial, political and philosophical debate to contemplate a world where cancer no longer kills.

[Uhohinc]

https://www.medpagetoday.com/genetics/generalgenetics/53643?pop=0&ba=1&xid=tmd-md&hr=trendMD
British apply for first designer embryo research in a human, with reference to Chinese research

[Uhohinc]

From Endpoints:
→ David Meeker left Genzyme last April after a 23-year run, including six years as president after Sanofi stepped in to buy the landmark biotech six years ago. Five months later, after adding a string of biotech board positions to his schedule, Meeker has landed his next big gig in biotech running KSQ Therapeutics in Cambridge, MA. At KSQ, he says, you can start an experiment using cutting edge technology and no bias about outcomes. With CRISPR,“we can study all 20,000 genes in the genome across a multitude of disease models and find out which of those targets has the biggest impact in modulating the disease. We can do it one shot, 20,000 genes at a time.”

[Uhohinc]

http://bgr.com/2017/10/24/crispr-pigs-gene-editing-gmo-bacon/

Bacon is love, bacon is life, and now scientists want to make it even better (?). New research using the groundbreaking CRISPR gene editing process has led to pigs which are genetically tweaked to hold less body fat, and which stay warmer in cooler temperatures. Yes, genetically engineered bacon low-fat bacon could be headed to your breakfast table in the not-so-distant future.

The research, which was conducted by an international group of researchers in both China and the UK, was published in the Proceedings of the National Academy of Sciences and may very well hint at the inevitable future of highly customized farm animals.

Using the CRISPR technique, the scientists added a gene known to be used in controlling body temperature to the embryos of piglets. The gene isn't normally present in pigs, and the researchers wanted to see if it would help piglets -- which don't have much body when they are young -- maintain a warmer body temperature. That, in turn, would help the animals survive with ease in cooler climates while potentially increasing lean meat production.

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A total of 13 attempts were made to make mature sow's pregnant with the modified offspring, and three of those ended up being successful. Between the three females, 12 male piglets were born, and as those pigs grew into adults, their body fat was, on average, 24% less than in an unmodified pig. These new pigs, which were able to stay warmer without as much fat thanks to faster metabolisms, appeared perfectly healthy.

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The ethics of genetically modifying animals are a topic of much debate, and there are already plenty of laws in place that would prevent pigs such as these from being used for food in many countries, including the United States. At the moment, the verdict is still out on whether genetic tweaking can lead to long-term health affects in various species, as well as any animal (like humans) who make a habit of eating them.

China, on the other hand, is a bit more lax with its guidelines for genetically modified food sources, though the researchers haven't said one way or the other whether pigs such as these could eventually make their way to farms.

[Uhohinc]

As we can see Crspr gene editing is most suited to an embryo whereby EVERY cell which number several trillion in an adult human is altered subsequent to the very first edit.

I think a therapeutic cure is in sight an eventuality for adults with EPP and here is why. EPP is really not a Dermatological disorder, that’s just where the major damage and symptom manifest. It’s a blood cell (erythrocytes) protein binding heme from a genetic orgin.

I foresee very plausible taking some of an adult EPP patients bone marrow stem cells with a needle and isolating them. Then Crspr gene edit a dozen or so to modify the dna and insert for the common gene variant lacking in EPP adult human blood. After the edit these blood stem cells can be nurtured in a bioreactor to replicate hundred million more stem cells identical with the EPP gene edited out fixed. Then these hundred million edited stem cells are re-injected back into the original host EPP adult patients marrow to become a part of that humans life genome till they die.

[Uhohinc]

Gene editing about to get a lot more powerful
Associated Press
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WASHINGTON (AP) — Scientists are altering a powerful gene-editing technology in hopes of one day fighting diseases without making permanent changes to people's DNA.

The trick: Edit RNA instead, the messenger that carries a gene's instructions.

"If you edit RNA, you can have a reversible therapy," important in case of side effects, said Feng Zhang of the Broad Institute of MIT and Harvard, a gene-editing pioneer whose team reported the new twist Wednesday in the journal Science.


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A genome editing technique called CRISPR has revolutionized scientific research. It's a biological cut-and-paste tool that lets researchers spot a gene defect inside living cells and use molecular "scissors" to snip that spot, either deleting, repairing or replacing the affected gene.

Researchers are using CRISPR to try to improve crops, develop malaria-resistant mosquitoes, grow transplantable organs inside animals, and develop treatments that one day may help genetic diseases such as sickle cell or muscular dystrophy.

There are challenges for medical use. Because a change to DNA is permanent, accidentally cutting the wrong spot could lead to lasting side effects.

And DNA repair is harder to achieve in certain cells, such as brain and muscle cells, than in others, such as blood cells — so targeting RNA may offer an important alternative, said University of California, San Diego, professor Gene Yeo, who wasn't involved in Wednesday's study. His team is creating its own RNA-targeting version of CRISPR.

Disease can occur when a genetic defect leaves cells making too little or too much of a particular protein, or not making it at all.

RNA, a cousin of DNA, carries the gene's instructions to start the protein-making process. Editing RNA's instructions should result in temporary fixes to abnormal protein production, Zhang explained. Because RNA degrades over time, the changes theoretically would last only as long as the therapy was used.

To starting figuring out how, researchers returned to nature.

CRISPR was adapted for use in mammalian cells from a system that evolved in bacteria, and uses as its molecular scissors an enzyme named Cas9. Zhang's team examined relatives in the Cas protein family and found one, Cas13, that could target RNA instead. The researchers engineered a Cas13 variety so it sticks to RNA instead of cutting it. They then fused on another protein to edit that spot and tested it in lab dishes.

The research is in its earliest stages, requiring more work before it even could be tested in animals.

But San Diego's Yeo, who is using a different Cas approach to target RNA, praised the competing work.

"It really tells us that many Cas proteins can truly bind RNA," he said. "The smart thing to do is to test a lot of them."

[Uhohinc]

‘It becomes part of your DNA’: Internal human gene-editing gets first trial https://www.rt.com/usa/409945-gene-editing-dna-experiment/

[Uhohinc]

http://www.bbc.com/news/world-us-canada-41990981

Biohacking by AIDS patient self injecting engineered plasmids antibodies to go after the c4 cells.

[Uhohinc]

https://www.sciencenews.org/article/crisprcas9-can-reverse-multiple-diseases-mice
This is even more significant of a discovery. In Crispr-Cas9 the research has centered on editing and inserting genes as learned from bacteria. Easier to edit one cell or an embryo zygot, or many stem cells and reintroduce to let them proliferate is key for now. But an adult human is made of trillions of cellls already with their dna as is.

This discovery looks like a huge game changer in that genes can be turned on or off and therefore produce a given protein or ramp down a protein. The possibilities go everywhere. As an examples, what if pancreatic cells could be turned on to produce insulin, or an mcr1 or tyr gene on, or telomerase proteins .

All pharmaceuticals basically in a shotgun effect are doing this with little accuracy or selectivity to just one gene or a series.

[Uhohinc]

http://www.foxnews.com/tech/2017/08/03/dna-breakthrough-scientists-repair-genes-in-human-embryos-to-prevent-inherited-diseases.html

Oregon gene editing to remove bad genes in embryos to lead to cure of thousands of genetic diseases inferred.

[Uhohinc]

https://www.usnews.com/news/news/articles/2017-12-28/in-a-milestone-year-gene-therapy-finds-a-place-in-medicine

If this works for Hunters Syndrome than it is more complicated but foreseeable feasible with some adapted approaches to work in the genes of EPP.

[Uhohinc]

https://gizmodo.com/doctors-perform-breakthrough-fda-approved-gene-therapy-1823954611

Not to unrelated to see the gene aberration in blood cells that leads to EPP to be correctioned like this. For now it would not be financially feasible to transfusion these reconditioned cells into an EPP patient as these erythrocytes die out naturally after about 90 days.

For EPP, my guess is the gene therapy will have to be in marrow stem cells, which is a slightly complex than these first breakthrough attempts in gene therapy.

[Uhohinc]

https://www.cbsnews.com/video/crispr-the-gates-scholars-seaweed/
Mainstream press USA 60 minutes as the most watched of any television program collectively for 50 years gives a very understandable view on CRSPR technology and its transformative aspect as the biggest breakthrough of the century

[Uhohinc]

https://endpts.com/gene-editing-stocks-get-bushwhacked-as-new-study-highlights-cancer-risks/

P53 gene, activation or dis regulation appears to complicate Crspr with cancerous propensities.

[Uhohinc]

https://www.newsmax.com/health/health-news/gene-therapy-hemophiliacs-cure-chronic-pain/2018/08/14/id/876946/ Today gene therapy in blood cells for hemophilia if when successful is not far in a leap of logic to gene therapy in EPP in blood cells........eventually any cell.

[Uhohinc]

https://endpts.com/the-broad-wins-another-perhaps-final-round-in-war-over-feng-zhangs-crispr-patents/ Patent law in drugs will always be relavent to Clinuvel. Meanwhile China has advanced long ago into human embryo. As bad as the rule of law is in USA and ambiguities, its a thousand times worse in China, and then bribery alledged often, and foreign business bias.

[Uhohinc]

https://endpts.com/in-a-landmark-first-chinese-scientist-claims-birth-of-genetically-modified-babies-and-all-hell-breaks-loose/

China does first designed baby.

[Uhohinc]

Genetically modified 'shortcut' boosts plant growth by 40%
By Matt McGrath
Environment correspondent
29 minutes ago
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tobacco plantsImage copyrightBRIAN STAUFFER
Image caption
These genetically modified tobacco plants were found to be 40% more productive
Scientists in the US have engineered tobacco plants that can grow up to 40% larger than normal in field trials.
The researchers say they have found a way of overcoming natural restrictions in the process of photosynthesis that limit crop productivity.
They believe the method could be used to significantly boost yields from important crops including rice and wheat.
The study has been published in the journal Science.
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GM crops have benefits - Princess Anne
Researchers are growing increasingly concerned about the ability of the world to feed a growing population in a time of serious climate change.
It's expected that agricultural demand will increase globally by 60-120% by the middle of this century compared to 2005. Increases in crop yields however are rising by less that 2% per annum, so there's likely to be a significant shortfall by 2050.
While the use of fertilisers, pesticides and mechanisation have boosted yields over the past few decades, their potential for future growth is limited.
Instead, scientists are increasingly looking to improving the process of photosynthesis as a way of increasing food productivity.
GM cropImage copyrightJAMES BALTZ/COLLEGE OF AGRICULTURAL
Image caption
Aerial view of the 2017 field trials. Researchers found that plants engineered with a synthetic shortcut are about 40% more productive.
While plants use the energy from sunlight to turn carbon dioxide and water into sugars that fuel the plant's growth, the chemical steps involved produce some toxic compounds that actually limit the potential of the crop.
These toxins are then recycled by the plant in a process called photorespiration - but this costs the plant precious energy that could have been used to increase yield.
In this study, researchers set out to developed a way around the photosynthesis glitch.
"We've tried three different biochemical designs with the aim of shortcutting this very energy expensive process," said lead author Dr Paul South with the US Agricultural Research Service.
"It's been estimated that in plants like soybeans, rice and fruit and vegetables, it can be a significant drag on yield by as much as 36%. We've tried to engineer this shortcut to make them more energy efficient - and in field trials this translated into a 40% increase in plant biomass."
One important aspect of the problem is that it becomes more prevalent at higher temperatures and under drought conditions.
"Our goal is to build better plants that can take the heat today and in the future, to help equip farmers with the technology they need to feed the world," said co-author Amanda Cavanagh, a postdoctoral researcher at the University of Illinois.
The researchers chose tobacco plants because they are easy and quick to modify. They also form a fully closed canopy in the field similar to many food crops.
The team is now hoping to use these findings to boost the yields of soybean, rice, potato and tomato plants.
GM cropImage copyrightL. BRIAN STAUFFER
Image caption
The experiment is significant say researchers because it involved two years of of field trials
"This process is very similar among all the crops that we are looking to grow," said Dr South.
"We are are really hoping that this is a technology that provides a tool that further optimises agriculture so that we are not using outside inputs as much and we are growing more food on less land."
However, the authors recognise that using genetic modification is controversial in many parts of the world.
They argue that a lengthy review process will ensure that if food crops are developed using this technology, they will be accepted by farmers and consumers alike.
"The research that's necessary to prove that it has low environmental impact and is safe for consumption takes a minimum of ten years and many more dollars in research funds to make sure that this is a good and safe food product," said Dr South.
The technology is being developed for royalty-free distribution to smallholder farmers in sub-Saharan Africa and in Southeast Asia.
It is being funded by the Bill & Melinda Gates Foundation, the Foundation for Food and Agriculture Research and the UK's Department for International Development.
Follow Matt on Twitter @mattmcgrathbbc.

[Uhohinc]

https://www.firstpost.com/tech/science/crispr-combined-with-new-art-treatment-cures-hiv-in-mice-for-first-time-6954381.html
Crspr cures mice of HIV virus.

[Uhohinc]

CRISPR gene editing will be used inside humans for the first time in treatment for blindness
By Rachael Rettner Senior Writer | LiveScience
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(Shutterstock)

The first study to test the gene-editing technology CRISPR inside the human body is about to get underway in the United States, according to news reports.

The study plans to use CRISPR to treat an inherited eye disorder that causes blindness, according to the Associated Press.

People with this condition have a mutation in a gene that affects the function of the retina, the light-sensitive cells at the back of the eye that are essential for normal vision. The condition is a form of Leber congenital amaurosis, one of the most common causes of childhood blindness that affects about 2 to 3 newborns out of every 100,000, according to the National Institutes of Health.

The treatment will correct the mutation using CRISPR, a tool that allows researchers to precisely edit DNA in a specific spot, the AP reported.

More From LiveScience

Associated Press
Leber congenital amaurosis
National Institutes of Health
CRISPR
Researchers will use an injection to deliver the treatment directly to the light-sensitive cells, according to a statement from Editas Medicine, the company that is conducting the study along with Allergan.

Researchers will use an injection to deliver the treatment directly to the light-sensitive cells, according to a statement from Editas Medicine, the company that is conducting the study along with Allergan.

The trial will enroll a total of 18 patients, both children (ages 3 and up) and adults.

The new study is different from the controversial research of a Chinese scientist who used CRISPR to edit the genomes of twin babies last year. In that case, the Chinese scientist edited the DNA of embryos, and these gene alterations could be passed down to the next generation, the AP reported. In the new study, the DNA edits made in the children and adults cannot be passed down to their offspring, the AP said.

10 Amazing Things Scientists Just Did with CRISPR
Unraveling the Human Genome: 6 Molecular Milestones
Bionic Humans: Top 10 Technologies

[Uhohinc]

This has rudiments in conceptualization to be applicable to EPP. And researchers in the area of numerous blood gene disorders I think are chomping at the bit to move Crspr into the marrow progenitor blood cells . And thats where most (but not all) EPP patients defect in heme synthesis begins.

Concepts usually take a long time if they ever happen.........And from my first post above it looks like its been 4years since Crspr made its headlines debut.

I would have thought 4 years ago we would be where this study is now about 2 years ago.......So with no big complications there is ? years to go yet.

And Crspr editing embryologically for EPP ..................has ethics to overcome, then the improvements in technovations, then i think it will be a screening of dna of a embryo a few weeks after fertilization for thousands of genetic abberations, which will be an abort well before an gene edit, and that will just start the long slow attrition of EPP patients whom never are born.

Years away to effect Clinuvel.

[Uhohinc]

https://www.sciencedirect.com/science/article/pii/S0959437X19300309

And here come designer colored aquarium fish...

[Uhohinc]

https://www.google.com/search?client=safari&channel=ipad_bm&biw=1024&bih=638&tbs=qdr%3Ad&ei=M24JXqqSGdGv-gSvmZj4Bg&q=60+minutes&oq=60+minutes&gs_l=mobile-gws-wiz-serp.3..46i131i275j0i131l3j0i3j0i131l3.25814.29850..30821...0.1..0.197.1121.3j7......0....1.......1..0i71j41j0i273j46i131j46j0j46i273i275j19.mzRrUqaJlN0

Sickle cell disease NIH funded study of gene therapy appearing to deliver very good results in patients. And this 60 minutes has been on USA television over 50 years with top ten most viewed.

Significant in that Sickle cell is one gene defect the blood cells, as are all the porphyrias.

Its easier to say that its not if EPP can be cured, but when.
With this high profile news on 60 minutes and so many one gene disorders, the US government NIH will pour billions if not unlimited funding into gene therapy.

https://www.google.com/search?client=safari&channel=ipad_bm&biw=1024&bih=638&tbs=qdr%3Ad&ei=M24JXqqSGdGv-gSvmZj4Bg&q=60+minutes&oq=60+minutes&gs_l=mobile-gws-wiz-serp.3..46i131i275j0i131l3j0i3j0i131l3.25814.29850..30821...0.1..0.197.1121.3j7......0....1.......1..0i71j41j0i273j46i131j46j0j46i273i275j19.mzRrUqaJlN0

The psychodellic based mushroom video is worth watching also.

[Uhohinc]

Very odd in the above there are deleted posts..........not by me.

[Uhohinc]

https://www.techtimes.com/articles/241316/20190412/chinese-experiment-creates-super-monkeys-with-human-brain-genes.htm

Chinese lab starting Planet of the Apes

[Uhohinc]

https://www.nature.com/articles/d41586-019-03919-0

[Uhohinc]

https://futurism.com/the-byte/sean-parker-chides-elon-musk
Clinuvel investor Sean Parker sees genetic edits manipulation as a bigger and closer at hand threat more than Elon Musk artificial intelligence threat.

Musk apparently has a evolving technological plan, 5 year initially to 25 year, augmentation to complete integration, of human brain to artificial intelllgence.

Future sharecafe posters of tomorrow land will be so smart, they will not consider communicating with people of today......................it would be like us communicating with a tree.

[Uhohinc]

https://en.as.com/en/2020/08/03/latest_news/1596459547_022260.html

Claim from senior Chinese researcher whom worked at Wahuan Chinese Military VirusLab that the Covid virus created in that lab from a template for genetic modifacations.

This Covid, never before seen , behaves like no other known vaccine.

[Uhohinc]

https://www.nature.com/articles/s41591-020-0820-9 Counter to the above claim of Chinese military gene engineering seems to be more plausible,  there was no evidence or facts to check from the Chinese doctor.

But the Chinese government is secretive (of everything)

[Uhohinc]

NOVEMBER 18, 2020

Revolutionary CRISPR-based genome editing system treatment destroys cancer cells

by  Tel Aviv University

Researchers at Tel Aviv University (TAU) have demonstrated that the CRISPR/Cas9 system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer. The researchers developed a novel lipid nanoparticle-based delivery system that specifically targets cancer cells and destroys them by genetic manipulation. The system, called CRISPR-LNPs, carries a genetic messenger (messenger RNA), which encodes for the CRISPR enzyme Cas9 that acts as molecular scissors that cut the cells' DNA.

The revolutionary work was conducted in the laboratory of Prof. Dan Peer, VP for R&D and Head of the Laboratory of Precision Nanomedicine at the Shmunis School of Biomedicine and Cancer Research at TAU. The research was conducted by Dr. Daniel Rosenblum together with Ph.D. student Anna Gutkin and colleagues at Prof. Peer's laboratory, in collaboration with Dr. Dinorah Friedmann-Morvinski from the School of Neurobiology, Biochemistry & Biophysics at TAU; Dr. Zvi R. Cohen, Director of the Neurosurgical Oncology Unit and Vice-Chair of the Department of Neurosurgery at the Sheba Medical Center; Dr. Mark A. Behlke, Chief Scientific Officer at IDT Inc. and his team; and Prof. Judy Lieberman of Boston Children's Hospital and Harvard Medical School.

The results of the groundbreaking study, which was funded by ICRF (Israel Cancer Research Fund), were published in November 2020 in Science Advances.

"This is the first study in the world to prove that the CRISPR genome editing system can be used to treat cancer effectively in a living animal," said Prof. Peer. "It must be emphasized that this is not chemotherapy. There are no side effects, and a cancer cell treated in this way will never become active again. The molecular scissors of Cas9 cut the cancer cell's DNA, thereby neutralizing it and permanently preventing replication."

To examine the feasibility of using the technology to treat cancer, Prof. Peer and his team chose two of the deadliest cancers: glioblastoma and metastatic ovarian cancer. Glioblastoma is the most aggressive type of brain cancer, with a life expectancy of 15 months after diagnosis and a five-year survival rate of only 3%. The researchers demonstrated that a single treatment with CRISPR-LNPs doubled the average life expectancy of mice with glioblastoma tumors, improving their overall survival rate by about 30%. 

Ovarian cancer is a major cause of death among women and the most lethal cancer of the female reproductive system. Most patients are diagnosed at an advanced stage of the disease when metastases have already spread throughout the body. Despite progress in recent years, only a third of the patients survive this disease. Treatment with CRISPR-LNPs in a metastatic ovarian cancer mice model increased their overall survival rate by 80%.

"The CRISPR genome editing technology, capable of identifying and altering any genetic segment, has revolutionized our ability to disrupt, repair or even replace genes in a personalized manner," said Prof. Peer. "Despite its extensive use in research, clinical implementation is still in its infancy because an effective delivery system is needed to safely and accurately deliver the CRISPR to its target cells. The delivery system we developed targets the DNA responsible for the cancer cells' survival. This is an innovative treatment for aggressive cancers that have no effective treatments today."

The researchers note that by demonstrating its potential in treating two aggressive cancers, the technology opens numerous new possibilities for treating other types of cancer as well as rare genetic diseases and chronic viral diseases such as AIDS.

"We now intend to go on to experiments with blood cancers that are very interesting genetically, as well as genetic diseases such as Duchenne muscular dystrophy," says Prof. Peer. "It will probably take some time before the new treatment can be used in humans, but we are optimistic. The whole scene of molecular drugs that utilize messenger RNA (genetic messengers) is thriving—in fact, most COVID-19 vaccines currently under development are based on this principle. When we first spoke of treatments with mRNA twelve years ago, people thought it was science fiction. I believe that in the near future, we will see many personalized treatments based on genetic messengers—for both cancer and genetic diseases. Through Ramot, the Technology Transfer Company of TAU, we are already negotiating with international corporations and foundations, aiming to bring the benefits of genetic editing to human patients."

[Uhohinc]

https://www.sciencemag.org/news/2020/12/crispr-and-another-genetic-strategy-fix-cell-defects-two-common-blood-disorders

As EPP, and all porphyrias are also one gene disorders in blood, so is sickle cell and this Thesilia or such.....

This is VERY good news for EPP sufferors, VERY bad news for Clinuvel revenues in EPP.

[Uhohinc]

1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive

December 15, 20205:02 AM ET

Heard on Morning Edition

ROB STEIN

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Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says.

Victoria Gray

The last thing a lot of people want to do these days is get on a plane. But even a pandemic would not stop Victoria Gray. She jumped at the chance to head to the airport this summer.

"It was one of those things I was waiting to get a chance to do," says Gray.

She had never flown before because she was born with sickle cell disease. She feared the altitude change might trigger one of the worst complications of the devastating genetic disease — a sudden attack of excruciating pain.

But Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.

SHOTS - HEALTH NEWSA Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving

About a year after getting the treatment, it was working so well that Gray felt comfortable flying for the first time. She went to Washington, D.C., to visit her husband, who has been away for months on deployment with the National Guard.

"It was exciting. I had a window. And I got to look out the window and see the clouds and everything," says Gray, 35, of Forest, Miss.

Gray wore a mask the whole time to protect herself against the coronavirus, kept her distance from other people at the airport, and arrived happily in Washington, D.C., even though she's afraid of heights.

"I didn't hyperventilate like I thought I would," Gray says, laughing as she recounts the adventure in an interview with NPR.

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NPR has had exclusive access to follow Gray through her experience since she underwent the landmark treatment on July 2, 2019. Since the last time NPR checked in with Gray in June, she has continued to improve. Researchers have become increasingly confident that the approach is safe, working for her and will continue to work. Moreover, they are becoming far more encouraged that her case is far from a fluke.

At a recent meeting of the American Society for Hematology, researchers reported the latest results from the first 10 patients treated via the technique in a research study, including Gray, two other sickle cell patients and seven patients with a related blood disorder, beta thalassemia. The patients now have been followed for between three and 18 months.

All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they've had to undergo before getting the billions of edited cells infused into their bodies.

The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.

"I'm very excited to see these results," says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR. "Patients appear to be cured of their disease, which is simply remarkable."

Another nine patients have also been treated, according to CRISPR Therapeutics in Cambridge, Mass., and Vertex Pharmaceuticals in Boston, two companies sponsoring the research. Those individuals haven't been followed long enough to report any results, officials say.

But the results from the first 10 patients "represent an important scientific and medical milestone," says Dr. David Altshuler, Vertex's chief scientific officer.

The treatment boosted levels of a protein in the study subjects' blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with. Hemoglobin is necessary for red blood cells to carry oxygen.

Analyses of samples of bone marrow cells from Gray six months after getting the treatment, then again six months later, showed the gene-edited cells had persisted the full year — a promising indication that the approach has permanently altered her DNA and could last a lifetime.

"This gives us great confidence that this can be a one-time therapy that can be a cure for life," says Samarth Kulkarni, the CEO of CRISPR Therapeutics.

Gray and the two other sickle cell patients haven't had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she'd needed most of her life.

Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven't needed the regular blood transfusions that had been required to keep them alive.

"It is a big deal because we we able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life," says Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville. Frangoul is Gray's doctor and is helping run the study.

For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth.

The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.

"It is opening the door for us to show that this therapy can not only be used in sickle cell and thalassemia but potentially can be used in other disorders," Frangoul says.

Doctors have already started trying to use CRISPR to treat cancer and to restore vision to people blinded by a genetic disease. They hope to try it for many other diseases as well, including heart disease and AIDS.

The researchers stress that they will have to follow Gray and many other patients for a lot longer to be sure the treatment is safe and that it keeps working. But they are optimistic it will.

Gray hopes so too.

"It's amazing," she says. "It's better than I could have imagined. I feel like I can do what I want now."

The last year hasn't always been easy for Gray, though. Like millions of other Americans, she has been sheltering at home with three of her children, worrying about keeping them safe and helping them learn from home much of the time.

"I'm trying to do the things I need to do while watch them at the same time to make sure they're doing the things they need to do," Gray says. "It's been a tough task."

But she has been able do other things she never got to do before, such as watch her oldest son's football games and see her daughter cheerleading.

"This is really a life-changer for me," she says. "It's magnificent."

She's now looking forward to going back to school herself, learning to swim, traveling more when the pandemic finally ends, and watching her children grow up without them worrying about their mother dying.

"I want to see them graduate high school and be able to take them to move into dorms in college. And I want to be there for their weddings — just everything that the normal people get to do in life. I want to be able to do those things with my kids," she says. "I can look forward now to having grandkids one day — being a grandmama."

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[Kenneth Steele]

I know the Patient one for the first phase trials

Sent from my iPhone

On Dec 15, 2020, at 12:26 PM, Uhohinc <MichaelBa...@gmail.com> wrote:



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[Kenneth Steele]

It work’s 

Sent from my iPhone

On Dec 15, 2020, at 11:48 PM, Kenneth Steele <kst...@alconre.com> wrote:

I know the Patient one for the first phase trials

[Uhohinc]

Researchers invent new gene-editing tool

by Sharon Parmet, University of Illinois at Chicago

A double stranded DNA fragment. Credit: Vcpmartin/Wikimedia/ CC BY-SA 4.0

Researchers from the University of Illinois Chicago have discovered a new gene-editing technique that allows for the programming of sequential cuts—or edits—over time.

CRISPR is a gene-editing tool that allows scientists to change the DNA sequences in cells and sometimes add a desired sequence or genes. CRISPR uses an enzyme called Cas9 that acts like scissors to make a cut precisely at a desired location in the DNA. Once a cut is made, the ways in which cells repair the DNA break can be influenced to result in different changes or edits to the DNA sequence.

The discovery of the gene-editing capabilities of the CRISPR system was described in the early 2010s. In only a few years, scientists became enamored with the ease of guiding CRISPR to target almost any DNA sequence in a cell or to target many different sites in a cell in a single experiment.

"A drawback of currently available CRISPR-based editing systems is that all the edits or cuts are made all at once. There is no way to guide them so that they take place in a sequential fashion, one after the other," said UIC's Bradley Merrill, associate professor of biochemistry and molecular genetics at the College of Medicine and lead author of the paper.

Merrill and colleagues' new process involves the use of special molecules called guide RNA that ferry the Cas9 enzyme within the cell and determine the precise DNA sequence at which Cas9 will cut. They call their specially engineered guide RNA molecules "proGuides," and the molecules allow for the programmed sequential editing of DNA using Cas9.

Their findings are published in the journal Molecular Cell.

While proGuide is still in the prototype phase, Merrill and colleagues plan to further develop their concept and hope that researchers will be able to use the technique soon.

"The ability to preprogram the sequential activation of Cas9 at multiple sites introduces a new tool for biological research and genetic engineering," Merrill said. "The time factor is a critical component of human development and also disease progression, but current methods to genetically investigate these processes don't work effectively with the time element. Our system allows for gene editing in a pre-programmed fashion, enabling researchers to better investigate time-sensitive processes like how cancer develops from a few gene mutations and how the order in which those mutations occur may affect the disease."

Explore further

Biochemists discover cause of genome editing failures with hyped CRISPR system