An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.
Researchers recruited 14 people with Leber Congenital Amaurosis (LCA), a rare genetic condition that causes babies to lose some or all of their sight from birth.
Eleven of the 14 had measurable improvements in the vision of one eye that received a single injection of a gene-editing medication, researchers report in the New England Journal of Medicine.
