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The JAMA Forum
November 12, 2019

Heritable Genome Editing—Edited Eggs and Sperm to the Rescue?

Author Affiliations
  • 1Professor of Medical Science and the former dean of Medicine and Biological Sciences at the Warren Alpert Medical School of Brown University in Providence, Rhode Island
  • 2James A. Attwood and Leslie Williams professor of Law and director of the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School, Cambridge, Massachusetts
JAMA. 2019;322(18):1754-1755. doi:10.1001/jama.2019.17538

“[We] might anticipate the in vitro culture of germ cells … coupled with recognition, selection and integration of the desired genes…” Nobel Laureate Joshua Lederberg, PhD (1963)

Heritable genome editing is widely predicted to render inborn afflictions a thing of the past. Topping the list of edit-worthy maladies are single-gene disorders for which preimplantation genetic diagnosis is unworkable. In addition, an insufficient number of viable embryos without the disease mutation is an important limitation in preimplantation genetic diagnosis, and in such cases, heritable genome editing might offer an alternative strategy.

Constraints along these lines have frequently undermined some families’ attempts to have a baby—a “savior sibling”—who could serve as a stem cell donor to a sick older sibling who might benefit. Heritable genome editing could also be brought to bear on disease-predisposing gene variants, such as a variant of the APOE gene that contributes to Alzheimer disease risk; a variant of the LPA gene that contributes to atherosclerotic cardiovascular disease; a variant of the MYPBC3 gene that causes hypertrophic cardiomyopathy; and variants in BRCA genes that increase breast and ovarian cancer risk. Currently, the focus of preclinical research, with safety and efficacy in mind, heritable genome editing remains years away from the clinic.