[HTML][HTML] rAAV-mediated cochlear gene therapy: prospects and challenges for clinical application

F Blanc, M Mondain, AP Bemelmans, C Affortit… - Journal of Clinical …, 2020 - mdpi.com
F Blanc, M Mondain, AP Bemelmans, C Affortit, JL Puel, J Wang
Journal of Clinical Medicine, 2020mdpi.com
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 …
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.
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