Gene Therapy for Inherited Retinal Dystrophies: From Bench to Bedside

The chapter reviews the scientific basis, clinical development, and therapeutic outcomes of gene therapy approaches for inherited retinal dystrophies, with particular focus on subretinal and intravitreal delivery strategies. It critically appraises landmark clinical trials, discusses the expanding pipeline of gene replacement and editing therapies targeting conditions such as Leber congenital amaurosis, choroideremia, and X-linked retinitis pigmentosa, and addresses outstanding challenges in long-term efficacy and accessibility.