Gene Therapy

Our laborartory has a long-standing interest in genetic hearing loss. The Laboratory has been involved in cloning and characterizing numerous deafness genes. Though more than one hundred genetic deafness genes have been identified, still no therapy is currently available. The next frontier in genetic hearing loss is the development of treatment. The Chen lab has recently shown that proteins can be directly delivered into the mammalian inner ear in vivo with functional consequences. Our lab has used protein delivery to demonstrate genome editing in the inner ear hair cells by CRISPR/Cas9 technology, and we are now working on combining protein deliveryand genome editing as a potential new treatment for genetic hearing loss. This combinatory approach should open the doors to the efficient study of virtually all genes in the inner ear.