Lancet
-
Multicenter Study
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.
Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 (REP1). We assessed the effects of retinal gene therapy with an adeno-associated viral (AAV) vector encoding REP1 (AAV.REP1) in patients with this disease. ⋯ UK Department of Health and Wellcome Trust.