The Promise and Perils of Gene Editing

Few things evoke a greater sense of desperation than a parent seeking an experimental treatment for their child. That desperation can make even extraordinary risks seem worth taking. This dynamic has come into stark relief after Mei, a 6-year-old girl, died of complications from an experimental gene-editing therapy administered in China that her parents aggressively pursued to treat her developmental disabilities. Their story, detailed in a recent investigation by Science and Retraction Watch, is a tragic reminder of what is at stake.

Gene-editing therapies, like the one received by Mei, involve modifying genes in non-reproductive cells to treat diseases. Recent successes have made their promise increasingly tangible. In 2023, exagamglogene autotemcel (Casgevy) -- a treatment for sickle cell disease -- became the first gene-editing therapy approved by the FDA. More recently, Baby KJ received a bespoke (highly personalized) base editing therapy for a rare and severe metabolic disease. The treatment was safe and well-tolerated, and after three doses, Baby KJ continues to thrive.

Yet somatic gene-editing treatments are not without risk. CRISPR/Cas9-based gene-editing can produce off-target (unintended) edits...