Viral vectors for gene delivery to the inner ear

Abstract

Gene therapy using virus vectors to treat hereditary diseases has made remarkable progress in the past decade. There are FDA-approved products for ex-vivo gene therapy for diseases such as immunodeficiencies (e.g., SCID), and in vivo gene therapy for a rare blindness and neuro-muscular disease. Gene therapy for hereditary hearing loss has picked up pace in the past five years due to progress in understanding disease gene function as well as the development of better technologies such as adeno-associated virus (AAV) vectors, to deliver nucleic acid to target cells in the inner ear. This review has two major goals. One is to review the state of the art for investigators already working in preclinical cochlear gene therapy. The other is to present the language of vectorology and important considerations for designing and using AAV vectors to inner ear neurobiologists who might use AAV vectors in the cochlea for either therapeutic or basic biological applications.