Can Gene Therapy Halt Diseases in Babies Before They’re Even Born?

July 27, 2018

(STAT News) – Gene therapies — in which a corrective gene hitches a ride on a virus into a patient’s cells — are being tested as potential cures or treatments for sickle cell, Duchenne muscular dystrophy, and a range of other diseases. Some patients have already been treated with a gene therapy for an inherited form of blindness, the first to be approved in the U.S. Now, imagine the virus-gene rig slipping into a pregnant woman’s womb, where it would reach the fetus and start remedying a disease before the baby is even born. That kind of procedure remains an untested goal for now — one whose feasibility has been debated by scientists for years. But advances in fetal gene therapies in animal models have raised the real possibility that clinical trials may be soon at hand.