Gene Therapy Helps Young Patient Hear
An eleven-year-old boy who was born deaf in both ears now only has mild to moderate hearing loss in one ear after undergoing an experimental gene therapy for hereditary hearing loss.
This is the first time a gene therapy has been used as a potential treatment for hereditary hearing loss in the United States.
The treatment was conducted by Children’s Hospital of Philadelphia (CHOP) in October 2023 and targeted otoferlin gene (OTOF)-mediated hearing loss. During the procedure, gene therapy containing copies of the OTOF gene was placed in the patient’s inner ear.
“Gene therapy for children with OTOF-mediated hearing loss has been designed to restore hearing by delivering copies of normal OTOF genes into the inner ear. With normal OTOF genes, the sensory cells will be able to function so they can respond to sound and activate the auditory nerve to send impulses to the brain,” an article on CHOP’s website stated. “Functioning OTOF genes are encased in a viral vector, a modified form of a non-disease-causing virus, which allows them to be delivered into cells of the cochlea in the inner ear. The vector solution is directly injected into the internal fluid of the cochlea during the procedure.”
Approximately 1 in 500 infants are affected by genetic hearing loss.
CHOP is one of several clinical trial sites in the world participating in the trial, which is sponsored by Akouos, Inc., and complete in collaboration with the Clinical In Vivo Gene Therapy group at CHOP. To learn more, view the study.
