Gene Replacement

Replacement, Editing, and the Two Routes Into the Eye: How Gene Therapy Is Being Built for Inherited Retinal Diseases

Gene therapy for inherited retinal diseases employs two main strategies: gene replacement or gene editing, delivered via subretinal or intravitreal injection. While Luxturna offers a successful model for LCA, ongoing research for conditions like achromatopsia highlights the complexities and challenges in developing effective treatments.