Gene therapy for Leber congenital amaurosis (LCA) delivers a functional copy of a mutated gene to retinal cells, usually using an adeno-associated virus (AAV) vector injected under the retina. The first FDA-approved treatment, Luxturna, targets RPE65 mutations and has shown improved night vision and visual fields in many patients.
- Process: A tiny surgical incision allows subretinal injection of the viral vector carrying the healthy gene.
- Outcome: Can restore some visual function, especially in dim light, but not full sight.
- Limitations: Effective only for certain mutations; benefits vary, and it’s not a cure.
- Risks: Includes inflammation, retinal detachment, or increased eye pressure.
- Research: Ongoing trials for other genes like CEP290 and GUCY2D.