Gene therapy yields ‘modest’ impact on retinal sensitivity in Leber’s congenital amaurosis

Gene therapy resulted in a modest and temporary improvement in retinal sensitivity among patients with Leber’s congenital amaurosis, according to a study.In a phase 1/2 open-label trial, 12 patients with early-onset, severe retinal dystrophy caused by mutations in RPE65 underwent gene therapy with a recombinant adeno-associated virus 2/2 (rAAV2/2) vector, rAAV2/2.hRPE65p.hRPE65, carrying RPE65 complementary DNA.