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RAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application.

ABSTRACT: 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.

SUBMITTER: Blanc F

PROVIDER: S-EPMC7073754 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature

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rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application.

Blanc Fabian F Mondain Michel M Bemelmans Alexis-Pierre AP Affortit Corentin C Puel Jean-Luc JL Wang Jing J

Journal of clinical medicine 20200221 2

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 summa ...[more]

PMID: 32098144

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