Gene therapy for ophthalmic diseases focuses on treating inherited retinal disorders (IRDs) and other genetic conditions affecting vision. Techniques involve delivering therapeutic genes directly into retinal cells using viral vectors like adeno-associated viruses (AAVs) or lentiviruses. The goal is to restore or preserve vision by replacing defective genes, promoting cell survival, or modifying disease progression. Clinical trials have shown promising results,particularly for conditions like Leber congenital amaurosis and retinitis pigmentosa. Challenges include optimizing delivery to specific retinal layers, ensuring long-term gene
expression, and addressing immune responses. Ongoing research aims to expand treatment options and improve outcomes for patients with ophthalmic genetic disorders.
Tracks:
Vector Development and Optimization
Clinical Trials and Treatment Efficacy
Gene Editing Technologies
Combination Therapies and Enhancing Delivery