Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Status:
Completed
Trial end date:
2014-08-01
Target enrollment:
Participant gender:
Summary
The purpose of the study is to assess the safety and efficacy of the active substance
rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset
retinal degeneration associated with RPE65 mutation.