AAV6(3pMut)-GFAP-Ush1c-a1

AAV6(3pMut)-GFAP-Ush1c-a1 is an experimental gene therapy designed to treat visual loss associated with Usher Syndrome Type 1C (USH1C). It utilizes a modified adeno-associated virus serotype 6 (AAV6) capsid containing three surface mutations (T492V, Y705F, and Y731F) to enhance its ability to traverse the retinal inner limiting membrane (ILM) following intravitreal injection. The vector employs a glial fibrillary acidic protein (GFAP) promoter to specifically drive the expression of the Ush1c-a1 transgene (encoding the harmonin protein) within Müller glial cells. By restoring harmonin expression in Müller cells, which provide structural and trophic support to photoreceptors, the therapy aims to improve retinal function and visual behavior. It was developed through a collaboration involving researchers at Louisiana State University Health Sciences Center, the University of Florida, and McGill University.