Our laboratory, which is part of the NIDCD Otolaryngology Surgeon-Scientist Program (OSSP), is focused on developing gene therapy as a treatment option for hearing loss and dizziness. Some patients with hereditary hearing loss and dizziness have mutations involving genes critical for inner ear development. Our laboratory is developing ways to delivery gene therapy to the mammalian inner ear in vivo. We are currently testing whether the delivery of normal copies of the mutated genes into animal models of hereditary hearing loss can restore hearing and balance.
Genome editing (zinc finger nucleases, TALENs, and CRISPR/Cas) allows modification of the genome at a single-nucleotide level. This can potentially be used to correct the mutation in the affected genes and restore normal function. We are currently studying whether genome editing can be used as a form of gene therapy to restore hearing and balance in animal models.