Inherited retinal dystrophies affect over 2 million people worldwide, often leading to progressive vision loss with limited treatment options. This medical study investigates adeno-associated virus (AAV) gene therapy approaches for treating Leber congenital amaurosis (LCA) and Stargardt disease. We evaluated different AAV serotypes for retinal targeting efficiency and developed optimized delivery protocols using subretinal injection techniques. In vitro studies using patient-derived retinal organoids demonstrated successful gene delivery and restoration of protein function in 78% of LCA10-mutant cells and 65% of ABCA4-mutant cells. Safety assessments revealed minimal immunogenic responses and no off-target effects. Clinical translation pathways are outlined with regulatory considerations for advancing gene therapies from laboratory to patient applications.