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rAAV-mediated

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

February 21, 2020

https://www.mdpi.com/2077-0383/9/2/589

https://www.ncbi.nlm.nih.gov/pubmed/32098144?dopt=Abstract

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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

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.

PMID: 32098144 [PubMed]

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