Gene therapy delivers genetic material into cells to treat or prevent disease by correcting or compensating for faulty genes. In ophthalmology, it is mainly used for inherited retinal disorders.
How It Works
Viral vectors such as adeno‑associated virus (AAV) carry a healthy copy of a gene into retinal cells via subretinal or intravitreal injection. The cells then produce functional protein, aiming to slow degeneration or improve function.
- First approved eye gene therapies target specific RPE or photoreceptor gene defects
- Best outcomes occur when significant viable cells remain
- Therapy is usually one‑time, though long‑term durability is still being studied
- Patient selection depends on precise genetic diagnosis
Safety and Monitoring
Side effects can include inflammation, immune reactions, or off‑target effects, so careful follow‑up is essential. Registries and trials continue to evaluate long‑term results.
- Not all inherited retinal diseases currently have gene therapies
- Genetic counselling is an important part of the pathway
- Future platforms may use gene editing or optogenetics
- Access is typically through specialist centres with trial or commercial programmes
