U.S. National Academies’ Gene Editing Recommendations

On February 14^th, an expert committee assembled by the U.S. National Academies of Science and Medicine released a comprehensive set of recommendations on the ethical use of genomic editing technology.

The bottom line?

“Heritable germline genome editing trials must be approached with caution, but caution does not mean they must be prohibited.”

Previous scientific policy and ethics committees have concluded that genomic editing technology is not yet ready for use in altering the human germline and further discourse is necessary to understand its implications. Notably, the NAS/NAM report marks the first recommendation from any major scientific organization that heritable human germline editing should be permitted under certain circumstances.

When exactly should the human germline be altered?

In their report entitled, “Human Genome Editing: Science, Ethics, and Governance,” the committee makes it clear that safe and effective germline intervention is currently unrealistic. Furthermore, in the United States, the FDA is prohibited from reviewing any “research in which a human embryo is intentionally created or modified to include a heritable genetic modification.”

However, technical barriers are being overcome and the permissibility of germline editing clinical trials differs between countries. Thus, the committee provides a strict set of criteria that should eventually be met in order for germline editing to be considered justifiable. They recommend that germline modification should only be used to prevent serious diseases when the harmful mutations are well understood, reversing the mutations is not likely to have an adverse effect, and no reasonable alternative is available. Additionally, genomic editing should not be used for enhancement of any kind, heritable or otherwise. The full criteria are listed in this one-page explainer.

“I agree with this cautious approach at this stage of our understanding,” says IGI Executive Director Jennifer Doudna. A pioneer in the world of CRISPR genetic engineering, Dr. Doudna was also one of the first scientists to initiate a global ethical conversation about its potential applications. She deems the report “impressive” and appreciates that it “provides a clear and balanced roadmap for research applications of human germline editing.”

Anything else?

Another noteworthy section emphasizes the critical importance of public engagement. The report states that public education, transparent public policy debate, and continual reassessment of the risks and benefits of genomic editing are all crucial to “applying societal values” to regulatory oversight.

The study also details a list of ethical principles to be followed in governing human genome editing and concludes that existing regulatory systems are adequate to provide proper oversight of scientists performing basic laboratory research as well as those making therapeutic, non-heritable changes to DNA in somatic cells (all tissues but sperm, egg, and embryonic cells).

Read the full report or check out the four-page summary.