But he was condemned by many of his peers, with the experiment labeled “monstrous,” “unethical” and a “huge blow” to the reputation of Chinese biomedical research. Chinese scientist He Jiankui was jailed for three years in 2019 after announcing that twin girls had been born with modified DNA to make them resistant to HIV, which he had managed using the gene-editing tool CRISPR/Cas9 before birth. An associate professor at the Southern University of Science and Technology in Shenzhen at the time, he said that he was “proud” of the achievement. In terms of human health, we can divide that into two different categories. The aim of gene editing is to make a precise change in a DNA sequence while leaving the rest of the genome untouched. Yale Insights asked Dr. Greg Licholai, a biotech entrepreneur and a lecturer at Yale SOM, to explain CRISPR’s potential and dangers. What are some of the applications, in the somewhat reasonable, predictable future? There had been previous gene-editing technologies, such as viral gene editing, gene replacement, and those have developed over several decades. If it’s right, the authors of the original study were wrong. So as you probably know, our book of life is made of DNA. And all of these have got some pretty profound complications and risks. In the United States the FDA closely monitors safety of any investigational drug, and all CRISPR drugs intended to go into people would have to meet the same rigorous testing standards. As with any new technology, there could be scientific bumps in the road. Normally, the clinical trial process to test any new therapy requires several very well studied stages. One is taking cells out of the body, manipulating them in the laboratory—either removing a defective gene or adding and enhancing an ability to do something by turning on a gene or fixing a gene—and then putting those cells back in the body. Sign up to read our regular email newsletters. And in fact, the application of CRISPR to foods has already been done. One of the other places that this is being actively worked on is, again, in animals. Gene editing has been around for decades but it remained extremely difficult and expensive until the revolutionary CRISPR technique was discovered in 2013. They actually call them CRISPR mice, and they are already being used in the research community. So theoretically, it would be safe to be able to treat those patients without the theoretical concern of affecting germ lines and affecting gene drive. Some critics have called for the paper to be withdrawn. Some of these are quite rare, like Fabry disease, but some are more common, like cystic fibrosis, which is the most common genetic disease in Caucasians. The name actually refers to the way it interacts with DNA. One of the most fascinating things about CRISPR is how quickly everything is developed, so in less than 10 years since the initial descriptions and initial papers were written, this technology has just exploded. Similarly, the application of CRISPR to animals has already been done. said in 2016 when she was interviewed by CNN. Given that it’s moved as far as it has in 10 years, where do you expect it to move in another 5 or 10 years? “Everyone has agreed that it cannot be used for germline engineering. Now a paper posted online on 5 July has proposed a simple and more plausible explanation for the controversial results. The other category would be actually injecting something into the body which can edit people’s genes so that within their own tissues those genes can either be turned on or off. Can you expand on that, on what regulations they have and what that means in terms of their competition with companies in the U.S.? Severe Weather An example of toxic gain of function is disease called transthyretin in which a mutation causes a clumping up of different proteins. Both have gotten louder in the five years since the development of CRISPR, which allows for much more precise editing of genes than previously existing tools. It’s changing pretty fast. And particularly some very high-placed scientists, in the United States, for example, the former director of the National Institute of Health, have called for a self-imposed ethical moratorium on CRISPR until more is known. It’s been well known now for some years that our own immune system has the ability to fight cancer cells and essentially dissolve micro-tumors. And that leads to a disease called amyloidosis, where these proteins, which normally don’t stick together, because of this kink in them due to the mutation, they become very sticky. The ability to apply it to larger animals such as food animals is in the very near future. Are the risks to the patient? And the other thing that’s changing is the effect of this international competition. Or eradicate certain types of invasive plants by introducing some kind of genetic manipulation that gets passed on and, again, you take out that one particular species. The microbiome: How bacteria regulate your health. “Doudna and Charpentier showed that CRISPR works like a pair of scissors that can be targeted to cut specific DNA sequences, said Andrew Holland, an assistant professor in the Department of Molecular Biology and Genetics at Johns Hopkins School of Medicine. Initially in 2015 and ’16, the reports were that the experiments were negative, and at least the Chinese researchers had claimed that they were working with nonviable human embryos anyway. “I agree the two mice are indeed more likely to be closely related,” says geneticist Gaetan Burgio of the Australian National University, one of the many critics of the original paper. The shared mutations in the edited mice were nowhere near DNA sequences resembling the one were targeted for editing, Pinello and colleagues point out, so it’s far from clear why CRISPR would cause mutations in these same sites in two different mice. We should make sure that the level of international scientific regulation and cooperation continues so the scientific developments can continue but also ensure safety. CRISPR stands for clustered regularly interspaced short palindromic repeats — a repeated DNA sequence in genomes. In 2017, for the first time, scientists used CRISPR to repair a genetic mutation—one that could cause a heart defect—in an embryo. It’s too early to tell if it’s successful or not. Gene editing can be used to introduce desirable changes into plants and animals (and perhaps people too one day), and to treat a range of disorders in people. Don’t Use COVID-19 as an Excuse to Turn Away Skilled Immigrants, Companies Invest in Startups to Repair Weaknesses. Both farming with animals as well as farming with crops. You can’t make heritable changes to human DNA. Daily News & Weather Updates We think we know what we’re affecting if we manipulate one gene for that particular species. But doubts were raised about these claims from the very beginning, not least because it was a tiny study involving just three mice. And within that language, there are certain regions which code for genes, and those genes are incredibly important because those genes go on to make up everything about us. No, the treatment of most of those diseases, monogenetic diseases—things like cystic fibrosis, sickle-cell, beta thalassemia—those are not germline mutations. Some is around competition, with new companies being formed. Now, at the same time, reports came out of China that researchers had begun working on human embryos. The technique discovered by Emmanuelle Charpentier, the director at the Max Planck Institute for Infection Biology, and Jennifer A. Doudna, a biochemist at the University of California Berkeley, is known as CRISPR/Cas9. The drug development process is tightly regulated across the world. Humans manipulating the genetic code, and those manipulations get passed on generation to generation to generation. You mentioned that Chinese researchers are operating in different structure. Or to all of us? “Not doing so does a disservice to the field and leaves the misleading impression that the strong statements and recommendations found in their paper are adequately supported by the data presented.”. So there are parts of the scientific community that are very concerned and are trying to be very thoughtful about how to proceed and how to proceed safely. Scientists have called for a moratorium on human germline editing, while efforts are being made to better regulate use of the technology. Another is to create medications that can be infused, or in some cases, self-therapy—taking blood and certain cells out of a body, manipulating them with CRISPR, and then putting them back in. For example, it’s been used to grow rice that accumulates lower levels of potentially toxic heavy metals and create livestock with more desirable traits.