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
Molecular Consequences of BEST1 Gene Mutations in Canine Multifocal Retinopathy Predict Functional Implications for Human Bestrophinopathies
