AAV-mediated Gene Therapy

Benefits

Risks & contraindications

Comparisons

Evidence quality

Highlighted claims

• Gene therapy was the most frequently studied advanced approach because many IRDs are caused by specific genetic mutations. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review
• Surgery required to deliver gene therapy still carries risks, so safety outcomes are not risk-free. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review
• AAV gene therapy for RPE65-associated LCA showed significant improvements in visual acuity and full-field stimulus testing at 1 year, but these gains were not sustained at 2–3 years. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review
• Evidence for visual acuity improvement and adverse events in RPE65-associated LCA gene therapy was graded high, supporting strong confidence in efficacy and safety for those outcomes. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review
• Voretigene neparvovec (AAV2-mediated gene replacement) has produced durable visual improvements in RPE65-associated LCA when sufficient retinal cells remain. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review
• One gene therapy review found only minimal gains in visual acuity and mobility, but moderate improvements in full-field stimulus testing. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review
• AAV gene therapy demonstrated reassuring safety results and evidence of therapeutic effect across 80 clinical trials. — Advanced therapeutic approaches for inherited retinal diseases: an umbrella review