Novel CLIC5 Gene Therapy Uses AAV Technology to Treat Hearing Loss

Scientists at Tel Aviv University in collaboration with scientists at Harvard Medical School have developed a new gene therapy that restores hearing and balance in animals with mutations in a gene not previously targeted for this kind of treatment. The gene therapy, described in EMBO Molecular Medicine , targets the CLIC5 gene which maintains the structure and stability of sensory hair cells in the inner ear and employs an optimized AAV vector for delivery—self-complementary AAV (scAAV).

“The inner ear consists of two highly coordinated systems: the auditory system, which detects, processes, and transmits sound signals to the brain, and the vestibular system, which enables spatial orientation and balance,” said senior author Karen Avraham, PhD, dean of the Gray Faculty of Medical &