DFNB16 hearing loss is caused by mutations in the STRC gene, which encodes a protein of the sensory cells of the inner ear. In the United States, there are ~700 cases/year of DFNB16, making it the second most common form of hereditary deafness. The STRC coding sequence is 5325 bp, too large to package in a single AAV vector. Previous work with dual-AAV delivery to a Strc- mouse model produced only partial rescue of hearing. In our Strc- mouse, we will test a new method for dual-AAV delivery of a long coding sequence which fuses N- and C-terminal RNAs from each AAV to create full-length mRNA. If successful, it would create a therapy for DFNB16, and be proof-of-concept for dual-AAV delivery of other large proteins.
Funding
Funding Duration
July 1, 2025 - June 30, 2027