AAV-based gene therapy for Usher syndrome

Usher syndrome is a rare genetic disorder that results in a combination of hearing loss and visual impairment. There currently are no approved treatments for vision loss in Usher and our lab is interested in testing AAV-based gene therapy approaches. However, for some forms of Usher syndrome, the gene carrying the disease-causing mutation is too large for traditional gene therapy approaches. Our lab is exploring a split gene treatment, called a multi AAV approach, to test its therapeutic potential for Usher syndrome.