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May 4 — Gene therapy studies using CRISPR/Cas-9 gene editing technology won't necessarily need in-depth review by the Recombinant DNA Advisory Committee (RAC) under revised NIH guidelines, an agency official said May 4.
The application of CRISPR/Cas-9 technology to human germ lines presents a big challenge to societies worldwide, Marina O'Reilly, biotechnology program adviser at the National Institutes of Health's Office of Science Policy, said. The gene splicing technology allows scientists to very efficiently disable or remove genes associated with disease, but has generated some controversy as it can be used to alter genes in embryos that are passed on to subsequent generations.
A key question is: “How do we ensure that the science is done safety and ethically?” O'Reilly said. “Some of the gene editing applications have the capacity to have a big impact on public health and the environment.”
The NIH began using an updated version of its Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules on April 27 (10 LSLR 07, 4/1/16)).
The most significant change in the guidelines is the requirement that institutional review boards (IRB) and institutional biosafety committees (IBC) must first review gene therapy studies at their institutions and recommend to the NIH whether an in-depth look by the RAC should be required. Previously, every gene therapy study receiving federal funding had to have a RAC review.
Upon receiving study registration and review board recommendations, the NIH will conduct its own review of every study protocol involving recombinant or synthetic nucleic acid molecules, but usually won't require a full-blown RAC review, O'Reilly said.
“In most situations NIH will concur with the IBC,” O'Reilly said at the Harvard Yale Symposium on the Intersection of Science, Safety and Policy, which focused on synthetic biology and CRISPR.
“If the NIH says a RAC is needed, then it goes to RAC. Otherwise, the registration is completed,” she said.
The use of CRISPR/Cas-9 technology in a new vector won't necessarily trigger the need for a RAC review, O'Reilly said. If the target gene is similar to ones previously targeted, then NIH might not recommend a RAC review.
“There will be some judgment there,” she said. “There will be some going forward with this and learning as we go.”
The NIH will get a decision back to the principal investigator within 10 days, O'Reilly said.
Under the new guidelines, the NIH performs a RAC review only occasionally, on gene therapy studies that involve new techniques or new vectors, or are considered particularly risky in terms of health or environmental implications should something go awry, O'Reilly explained.
Any laboratory that conducts gene therapy research and receives government funding is required to follow the new guidelines, she said.
The guidelines had barely changed since they were first created in 1976. Since then, there has been an explosion of new gene therapy applications, O'Reilly said.
The Institute of Medicine, now called the National Academy of Medicine, recommended in 2013 that changes be made to the guidelines.
Especially with the advent of CRISPR/Cas-9 techniques, the guidelines were due for an update, O'Reilly said.
Speaking about the reviewd guidelines, however, an IRB director at the meeting told O'Reilly, “This is just goofy.” He declined to provide his name to Bloomberg BNA. “If NIH is going to look at it anyway, then cut the IBC's out of it. It's convoluted,” he said.
O'Reilly responded that the NIH is simply putting in place what the academy recommended. “It took us a long time to come up with a plan to implement the IOM recommendations,” she said.
The CRISPR/Cas-9 technology is widely used in laboratories, on animal and human cells. The NIH funded more than $160 million in research grants involving CRISPR/Cas-9 technology in 2015, O'Reilly said.
They are now on the brink of being used in human adults in an attempt to treat or even cure serious diseases, such as HIV and sickle-cell anemia, according to research reports in Nature and The New England Journal of Medicine.
A number of human clinical trials are underway in the U.S. involving gene therapy for HIV, according to the research reports.
It is also possible to apply gene editing to mitochondria cells, to make changes to genes in order to cure a disease, that is passed along to successive generations, according to the journals.
Gene editing techniques can also be used in embryos, in an attempt to cure diseases or create life.
“There is concern that this technology would be used for more than therapeutic purposes, [such as] for designer babies,” O'Reilly said.
The NIH won't fund gene editing studies involving human embryos, due to ethical and health concerns. But such research is allowed in Japan, Sweden, the U.K. and China, which announced in 2015 that it had edited DNA in four embryos, O'Reilly said (9 LSLR 497, 5/1/15).
The China research used CRISPR/Cas-9 techniques in the nucleus of cells in human embryos. China's announcement kicked up fears in the U.S. and worldwide about scientists' ability to make permanent changes to human embryos which could be passed on to future generations, according to research reports.
So far, 29 nations have banned gene editing of human embryos, O'Reilly said. The U.K. approved gene editing with human embryos earlier this year (10 LSLR 03, 2/5/16).
The National Academies of Sciences, Engineering and Medicine is studying the issue of gene editing in human embryos and expects to release a report later this year, O'Reilly said.
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