AAV-USH1C

AAV-USH1C is an adeno-associated virus (AAV) vector-based gene therapy designed to treat *Usher syndrome type 1C* by delivering a functional copy of the USH1C gene (encoding the protein harmonin) to affected cells[1][7]. The viral vector (often AAV2/Anc80L65) mediates long-term transgene expression in post-mitotic tissue, specifically targeting inner ear hair cells to restore auditory and vestibular function in animal models[1][7]. By introducing a normal USH1C gene, it compensates for loss-of-function mutations linked to the syndrome, enabling restoration of sensory cell structure and function. The therapy is under preclinical development, with multiple studies demonstrating successful gene replacement and functional recovery in animal (mouse) models for hearing and balance.