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Article Dans Une Revue Journal of Clinical Medicine Année : 2020

rAAV-mediated cochlear gene therapy: Prospects and challenges for clinical application

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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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hal-03521743 , version 1 (11-01-2022)

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Fabian Blanc, Michel Mondain, Alexis-Pierre Bemelmans, Corentin Affortit, Jean-Luc Puel, et al.. rAAV-mediated cochlear gene therapy: Prospects and challenges for clinical application. Journal of Clinical Medicine, 2020, 9 (2), pp.589. ⟨10.3390/jcm9020589⟩. ⟨hal-03521743⟩
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