Preliminary Results from an Adult Participant in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1Mutations
Consideration of Endpoints for the First Interventional Gene Therapy Clinical Study in Inherited Retinal Diseases Caused by BEST1 Mutations
Recombinant AAV-Mediated BEST1 Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Dependent Retinal Effects
