USH2A minigene therapy

USH2A minigene therapy is an investigational **AAV-delivered gene therapy** being developed for **USH2A-related inherited retinal diseases**, including **Usher syndrome type 2A** and **USH2A-associated autosomal recessive retinitis pigmentosa**. The approach uses **artificially shortened USH2A transgene constructs** designed to preserve enough usherin function while fitting within the packaging constraints of adeno-associated virus vectors, unlike the native full-length **USH2A** gene, which is too large for standard AAV delivery. It is intended as a **mutation-independent gene replacement strategy**, meaning it could potentially treat patients across different pathogenic USH2A variants by restoring production of a functional truncated usherin protein in retinal cells. The program originated from a sponsored research collaboration between **IVERIC bio** and **UMass Medical School**, and following IVERIC bio's acquisition, development is associated with **Astellas Gene Therapies**.

Other names

USH2A minigene therapyUSH-2A minigene therapyUSH 2A minigene therapymutation-independent USH2A minigene therapyUSH2A-related inherited retinal disease minigene therapyUSH-2A-related inherited retinal disease minigene therapyUSH 2A-related inherited retinal disease minigene therapy