Genetic disorder

Human gene editing therapies are OK in certain cases, panel advises

bubble boy
EDITING OUT DISEASE Gene therapy can cure a genetic disease called severe combined immunodeficiency, or “bubble boy,” disease. Using new gene editing techniques like CRISPR/Cas9 to treat genetic diseases is fine under certain conditions, but it should not be used to enhance people, a panel of experts says.

Human gene editing to prevent genetic diseases from being passed to future generations may be permissible under certain conditions, a panel of experts says.

Altering DNA in germline cells — embryos, eggs, and sperm, or cells that give rise to them — may be used to cure genetic diseases for future generations, provided it is done only to correct disease or disability, not to enhance people’s health or abilities, a report issued February 14 by the National Academies of Sciences and Medicine recommends. The decision contradicts earlier recommendations by organizers of a global summit on human gene editing, who concluded that gene editing with molecular scissors such as CRISPR/Cas9 should not be used to produce babies (SN: 12/26/15, p. 12).

Heritable gene editing is not yet ready to be done in people, says Alta Charo, a bioethicist at the University of Wisconsin‒Madison Law School who cochaired the panel. “We are not trying to greenlight heritable germline editing. We’re trying to find that limited set of circumstances where its use is justified by a compelling need and its application is limited to that compelling need,” says Charo. “We’re giving it a yellow light.”

National Academies reports carry no legislative weight, but do often influence policy decisions in the United States and abroad. It will be up to Congress, regulatory agencies such as the U.S. Food and Drug Administration, and state and local governments to implement the recommendations.

Supporters of new genetic engineering technologies hailed the decision.

“It looks like the possibility of eliminating some genetic diseases is now more than a theoretical option,” says Sean Tipton, a spokesman for the American Society for Reproductive Medicine in Washington, D.C. “That’s what this sets up.” Diseases such as cystic fibrosis and Huntington’s, which are caused by mutations in single genes, could someday be corrected by gene editing. More complex diseases or disorders caused by changes in multiple genes, such as autism or schizophrenia, probably would not be the focus of genome editing.

Others worry that…