NOVEL GENE THERAPY TECHNIQUE TO TREAT HEREDITARY DEAFNESS

Autosomal recessive deafness-9 (DFNB9), caused by mutations in the otoferlin gene (OTOF), is the most common form of hereditary deafness, accounting for 2-8% of all cases. Here, I review recent research on using dual adeno associated virus (AAV) mediated gene therapy to treat DFNB9 in a mouse model system. Dual AAV gene therapy repairs these mutations by injecting pairs of AAV vectors carrying separate fragments of Otoferlin DNA into the round window membrane to the affected cochlea. When these AAV vectors recombine, they produce the expression of the full-length gene and restores hearing. Dual AAV gene therapy provides a biologically regenerative treatment that is faster and less invasive than the cochlear implant currently used to treat DFNB9. This breakthrough will reshape the treatment of genetic diseases.