Gene therapy technique used to restore hearing in congenitally deaf children
The gene therapy technique used to restore hearing in congenitally deaf children involves the use of adeno-associated viruses (AAVs) to deliver a functional copy of the gene responsible for producing otoferlin, a protein necessary for the transmission of sound signals from the inner ear to the brain. This gene therapy approach targets children with a specific form of hereditary deafness caused by mutations in the OTOF gene, which encodes the otoferlin protein. In the clinical trials described, doctors inject the virus carrying the healthy gene directly into the inner ears of the children through a special surgical procedure. This allows the gene to be expressed in the cochlear cells, enabling these cells to produce the otoferlin protein, which is crucial for hearing. The therapy has shown significant success, with many of the treated children experiencing recovery of hearing and dramatic improvements in speech perception 1 2 4 5 6.
Sources:
- (1) Gene Therapy Brings Hearing to Kids With Congenital Deafness
- (2) Gene therapy restores hearing in children with hereditary deafness
- (3) Recent advances and future challenges in gene therapy for hearing ...
- (4) Experimental gene therapy helps treat kids with inherited hearing loss
- (5) Gene therapies restore hearing in several kids with inherited deafness
- (6) Gene Therapy Restores Hearing in Children ... - Mass Eye and Ear
- (7) Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time
- (8) Experimental Gene Therapy Enables Hearing in Five Children Born ...