rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application.
J Clin Med. 2020 Feb 21;9(2):
Authors: Blanc F, Mondain M, Bemelmans AP, Affortit C, Puel JL, Wang J
Over the last decade, pioneering molecular gene therapy for inner-ear disorders have achieved experimental hearing improvements after a single local or systemic injection of adeno-associated, virus-derived vectors (rAAV for recombinant AAV) encoding an extra copy of a normal gene, or ribozymes used to modify a genome. These results hold promise for treating congenital or later-onset hearing loss resulting from monogenic disorders with gene therapy approaches in patients. In this review, we summarize the current state of rAAV-mediated inner-ear gene therapies including the choice of vectors and delivery routes, and discuss the prospects and obstacles for the future development of efficient clinical rAAV-mediated cochlear gene medicine therapy.
PMID: 32098144 [PubMed]