A Chinese Court has Sentenced Dr He Jiankui to 3 years in Prison for Genetically Engineering Babies with CRISPR

A Chinese court sentenced the scientist who created the world’s first “gene-edited” babies to three years in prison on Monday, according to the official Xinhua media.

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The Russian Scientific Community Consensus on Genetically Engineering Humans

The Russian community of geneticists, clinicians and bioethicists have reached a consensus on the use of genome-editing technologies on human embryos and germ cells for clinical purposes. They consider that such experiments are premature at this point. Their view aligns with the position of the Russian ministry of health and sets the social context for further discussion of the technology.

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Russian Scientist has Started Editing Human Embryos

Russian biologist Denis Rebrikov has started gene editing in eggs donated by women who can hear to learn how to allow some deaf couples to give birth to children without the genetic mutation that impairs hearing. The news, detailed in an e-mail he sent to Nature on 17 October, is the latest in a saga that kicked off in June, when Rebrikov told Nature of his controversial intention to create gene-edited babies resistant to HIV using the popular CRISPR tool.

Rebrikov’s latest e-mail follows a September report in Russian magazine N+1 that one deaf couple had started procedures to procure eggs that would be used to create a gene-edited baby — but the eggs that Rebrikov has edited are from women without the genetic mutation that can impair hearing. He says the goal of the experiments is to better understand potentially harmful ‘off-target’ mutations, which are a known challenge of using CRISR–Cas9 to edit embryos.

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Researchers Appear to Have Cured HIV in a Mice With the Help of CRISPR

In a major collaborative effort, researchers at the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) have for the first time eliminated replication-competent HIV-1 DNA - the virus responsible for AIDS - from the genomes of living animals. The study, reported online July 2 in the journal Nature Communications, marks a critical step toward the development of a possible cure for human HIV infection.

"Our study shows that treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals," said Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Neuroscience, Director of the Center for Neurovirology, and Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine at Temple University (LKSOM). Dr. Khalili and Howard Gendelman, MD, Margaret R. Larson Professor of Infectious Diseases and Internal Medicine, Chair of the Department of Pharmacology and Experimental Neuroscience and Director of the Center for Neurodegenerative Diseases at UNMC, were senior investigators on the new study.

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A Third CRISPR Baby may Have Been Born

The Second International Summit on Human Genome Editing, held in Hong Kong last November, was meant to debate the pros and cons of genetically engineering humans. Instead, the proceedings were turned upside down by the revelation that He Jiankui, a Chinese biophysicist, had already done it.

He’d gone ahead and edited the DNA of twin girls with the powerful gene modification tool called CRISPR.

Then the Chinese scientist sprang a further surprise on the shocked gene-editing experts. A second Chinese woman, he said, was pregnant with yet another CRISPR baby. An early pregnancy test had confirmed it.

That third CRISPR baby is now due to be born at any moment—if he or she hasn’t come crying into the world already.

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A Chinese Researcher Claims to Have Produced First Genetically Engineered Babies,Twin Girls

A Chinese researcher claims that he helped make the world’s first genetically edited babies — twin girls born this month whose DNA he said he altered with a powerful new tool capable of rewriting the very blueprint of life.

If true, it would be a profound leap of science and ethics.

A U.S. scientist said he took part in the work in China, but this kind of gene editing is banned in the United States because the DNA changes can pass to future generations and it risks harming other genes.

Many mainstream scientists think it’s too unsafe to try, and some denounced the Chinese report as human experimentation.

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CRISPR COWS! CRISPR Used to Create Bovine Tuberculosis Resistant Cows

CRISPR/Cas9 gene-editing technology has been used for the first time to successfully produce live cows with increased resistance to bovine tuberculosis, reports new research published in the open access journal Genome Biology.

The researchers, from the College of Veterinary Medicine, Northwest A&F University in Shaanxi, China, used a modified version of the CRISPR gene-editing technology to insert a new gene into the cow genome with no detected off target effects on the animals genetics (a common problem when creating transgenic animals using CRISPR).

Dr Yong Zhang, lead author of the research, said: "We used a novel version of the CRISPR system called CRISPR/Cas9n to successfully insert a tuberculosis resistance gene, called NRAMP1, into the cow genome. We were then able to successfully develop live cows carrying increased resistance to tuberculosis. Importantly, our method produced no off target effects on the cow genetics meaning that the CRISPR technology we employed may be better suited to producing transgenic livestock with purposefully manipulated genetics."

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CRISPR has Been Used on People for the First Time

A team of Chinese scientists from Sichuan University in Chengdu have become the first to inject a person with cells modified with the gene-editing tool CRISPR-Cas9. The trial involved modifying a patient's own immune system cells to make them more effective at combating cancer cells and then injecting them back into the patient. The Chinese trial was approved back in July, and United States medical scientists also plan to use CRISPR as an experimental treatment for cancer patients in early 2017.

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China to Test CRISPR on Human Beings

A team of Chinese scientists will be the first in the world to apply the revolutionary gene-editing technique known as Crispr on human subjects.

Led by Lu You, an oncologist at Sichuan University’s West China hospital in Chengdu, China, the team plan to start testing cells modified with Crispr on patients with lung cancer in August, according to the journal Nature.

Crispr is a game-changer in bioscience; a groundbreaking technique which can find, cut out and replace specific parts of DNA using a specially programmed enzyme named Cas9. Its ramifications are next to endless, from changing the color of mouse fur to designing malaria-free mosquitoes and pest-resistant crops to correcting a wide swath of genetic diseases like sickle-cell anaemia in humans.

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CRISPR Gets Approval as Treatment for Cancer

An experimental cancer treatment that alters the DNA of patients has won a key approval to proceed with its first human tests using the controversial gene-altering tool known as Crispr.

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A CRISPR Tool for RNA Found

Researchers including Feng Zhang have confirmed that a bacterial protein hypothesized as a tool for targeted editing of RNA, similar to how CRISPR-Cas9 targets DNA, can indeed be used as an alternate editing approach. The finding holds important implications for a range of biological applications, such as marking, modifying and modulating RNA. Roughly half of all bacteria species utilize an immune system called the Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated genes (CRISPR-Cas), which protects the microbes from viruses and other invading DNA. While much focus in recent years has been directed at the ability to harness CRISPR-Cas systems to edit DNA, systems that specifically target RNA have been less studied. Here, Zhang and colleagues build on previous work to characterize the behavior of a new type of CRISPR-CAS system, C2c2, suspected to target RNA. Through a series of experiments, the researchers demonstrate that C2c2 can be used to cleave single-stranded RNA, but not double-stranded RNA; as well, it can be used to knockout messenger RNA of bacteria in vivo. In testing the ability of C2c2 to target specific RNA in Escherichia coli, the team found that it initially focuses on its target RNA, followed by a second phase in which it degrades RNA in a less specific way. Other RNA-targeting immune systems likely exist, the authors say, and further research will lead to the development of programmable molecular tools for in vivo RNA manipulation. One example includes modulating RNA function and translation, which could be used for large-scale screening of biological molecules, construction of synthetic regulatory circuits and other purposes, the authors say.

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CRISPR Cleared for Commericial Mushrooms

The US Department of Agriculture (USDA) will not regulate a mushroom genetically modified withthe gene-editing tool CRISPR–Cas9.

The long-awaited decision means that the mushroom can be cultivated and sold without passing through the agency's regulatory process—making it the first CRISPR-edited organism to receive a green light from the US government.

“The research community will be very happy with the news,” says Caixia Gao, a plant biologist at the Chinese Academy of Sciences’s Institute of Genetics and Developmental Biology in Beijing, who was not involved in developing the mushroom. “I am confident we'll see more gene-edited crops falling outside of regulatory authority.”

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In the age of CRISPR, Should Parents be Allowed to Edit Their Children's Genomes?

Many safety, technical and legal barriers still stand in the way of editing DNA in human embryos. But some scientists and ethicists say that it is important to think through the implications of embryo editing now — before these practical hurdles are overcome. What sort of world would these procedures create for those currently living with disease and for future generations?

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Massachusetts General Hospital Develops Improved CRISPR

A new engineered version of the gene-editing CRISPR-Cas9 nuclease appears to robustly abolish the unwanted, off-target DNA breaks that are a significant current limitation of the technology, reducing them to undetectable levels. In their report receiving advance online publication in Nature, Massachusetts General Hospital (MGH) researchers describe how altering the Cas9 enzyme to reduce non-specific interactions with the target DNA may greatly expand applications of the gene-editing technology.

"Our creation of a Cas9 variant that brings off-target effects to levels where we can no longer detect them, even with the most sensitive methods, provides a substantial advance for therapeutic applications in which you want to accurately hit your target without causing damage anywhere else in the genome," says J. Keith Joung, MD, PhD, associate chief for Research and the Jim and Ann Orr MGH Research Scholar in the MGH Department of Pathology, senior author of the Nature paper. "But its impact will also be incredibly important for research applications because off-target effects can potentially confound the results of any experiment. As a result, we envision that our high-fidelity variant will supplant the use of standard Cas9 for many research and therapeutic applications."

Used to create targeted DNA breaks at which genetic changes can be introduced, CRISPR-Cas9 nucleases combine a bacterial DNA-cutting enzyme called Cas9 with a short guide RNA sequence that can bind to the target DNA sequence. While easier to use than previous gene-editing tools, CRISPR-Cas9 nucleases have a well-characterized and significant limitation. As described in 2013 studies led by Joung and others, CRISPR-Cas9 nucleases can induce off-target DNA breaks at sites that resemble the on-target sequence. Subsequent investigations by Joung's team and others have reduced but never completely and consistently eliminated these off-target effects.

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CRISPR Used for First Time to Treat Genetic Disorder in Adult Mice

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy.

Researchers from Duke University had previously used CRISPR to correct genetic mutations in cultured cells from Duchenne patients, and other labs had corrected genes in single-cell embryos in a laboratory environment. But the latter approach is currently unethical to attempt in humans, and the former faces many obstacles in delivering treated cells back to muscle tissues.

Another approach, which involves taking CRISPR directly to the affected tissues through gene therapy techniques, also faces challenges, particularly with delivery. In the new study, Duke University researchers overcame several of these obstacles by using a non-pathogenic carrier called adeno-associated virus, or AAV, to deliver the gene-editing system.

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Stepping Toward Therapeutic use of CRISPR

Stepping toward therapeutic CRISPR

Authors:

Gagnon et al

Abstract:

Most new technologies for manipulating gene expression in mammalian cells are accepted at a relatively slow pace. Occasionally, however, a new technology is so robust and fills such a critical niche that its adoption is widespread and rapid. Fifteen years ago, duplex RNAs were such a technology. RNA interference (RNAi) in mammalian cells was first demonstrated in 2001 (1) and within 2 y RNAi was a commonly used tool throughout industry and academia. RNAi is making its way into clinical trials as a potential therapeutic as challenges in delivery to relevant tissues begin to be overcome (2–4).

Scientists are Working on Kill Switches for Genetically Modified Organisms

Gene editing has received a lot of press recently, thanks to a slew of recent advancements that allow scientists to do ever more with plant and animal genomes. Techniques, such as CRISPR-Cas9, are opening the doors to novel genetically modified crops (GMOs), stem cell research, synthetic biology and even experiments with human embryos.

However, with this explosion of research has come intense scrutiny. Modifying DNA, the blueprint of an organism, carries with it powerful implications and unknown risks. Just last week, an international summit on human gene editing was convened in Washington D.C. to discuss ethical guidelines for future gene editing research, among other agenda items. One of the biggest concerns about genetic modification is the potential for engineered microbes with altered DNA to escape the lab and wreak havoc on an unprepared world. Like Frankenstein’s monster, the fear is that these organisms may prove harmful to humans and difficult to stop. Fortunately, scientists have been working hard to ensure this nightmare scenario doesn’t occur.

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Beyond Extraordinary Claim: Harvard Prof Claims Aging Cure Within 5 Years

At the gene-editing summit, you can’t miss George Church. He’s the big guy with the bushy beard and wavy hair, someone who looks like he stepped out of an 18th century painting of “natural philosophers.” Church, who is 61, is among several hundred scientists, policymakers and thinkers on hand to discuss the powerful technology known as CRISPR, a new method for editing genes. The technique was invented in the past four years, and Church is among those who can claim at least partial credit for the innovation (there’s an intense legal battle over patents — a story for another day).

I mentioned to Church that this is the kind of work for which Nobels are awarded. He quickly responded that there are more important things in the balance than prizes. There are cures for human diseases, he said.

Church thinks that one of the ailments he can cure is aging. When I met him early this year, in his laboratory at Harvard Medical School, where he is professor of genetics, he expressed confidence that in just five or six years he will be able to reverse the aging process in human beings.

“A scenario is, everyone takes gene therapy — not just curing rare diseases like cystic fibrosis, but diseases that everyone has, like aging,” he said.

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Joint Conference in Washington, DC on the use of CRISPR-Cas9 on Humans

Don't look now, but the future just pulled into town.

Hundreds of scientists, policymakers and the president's science adviser have gathered Tuesday in Washington for what will be a three-day summit on genetic engineering, with a focus on a new, relatively simple technique for manipulating genes. It's fast and flexible, and just about anybody with some lab equipment and a little know-how can potentially alter the human species. The technique is called CRISPR-Cas9, or simply CRISPR, and more generically referred to as "gene editing."

The summit kicked off early Tuesday morning at the headquarters of the National Academy of Sciences, which is one of the sponsors, along with the National Academy of Medicine, the Royal Academy (Britain), and the Chinese Academy of Sciences. The Chinese scientists have been aggressive in using CRISPR, and one team made news this year when it reported results from experiments on nonviable human embryos.

“The overriding question is when, if ever, we will want to use gene editing to change human inheritance," summit chair David Baltimore of Caltech said in his introductory remarks.

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China is Using CRISPR to Rapidly Developing Genetically Customized Animals

China’s western Shaanxi Province is known for rugged windswept terrain and its coal and wool, but not necessarily its science. Yet at the Shaanxi Provincial Engineering and Technology Research Center for Shaanbei Cashmere Goats, scientists have just created a new kind of goat, with bigger muscles and longer hair than normal. The goats were made not by breeding but by directly manipulating animal DNA—a sign of how rapidly China has embraced a global gene-changing revolution.

Geneticist Lei Qu wants to increase goatherd incomes by boosting how much meat and wool each animal produces. For years research projects at his lab in Yulin, a former garrison town along the Great Wall, stumbled along, Qu’s colleagues say. “The results were not so obvious, although we had worked so many years,” his research assistant, Haijing Zhu, wrote in an e-mail.

That changed when the researched adopted the new gene-customizing technology called CRISPR–Cas9, a technique developed in the U.S. about three years ago. CRISPR uses enzymes to precisely locate and snip out segments of DNA, much like a word-processor finding and deleting a given phrase—a process known as “gene-editing.” Although it is not the first tool scientists have used to tweak DNA, it is by far more precise and cheaper than past technologies. The apparent ease of this powerful method now raises both tantalizing possibilities and pressing ethical questions.

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