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

1
INSERM - UMR 1051, Institut des Neurosciences de Montpellier, Hôpital Saint Eloi - Bâtiment INM, 80, rue Augustin Fliche - BP 74103, 34091 Montpellier, France
2
Université de Montpellier, 163 rue Auguste Broussonnet, 34090 Montpellier, France
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CHRU Montpellier—Centre Hospitalier Régional Universitaire, 34090 Montpellier, France
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Molecular Imaging Research Center, Institut de Biologie François Jacob, Direction de la Recherche Fondamentale, CEA, 92265 Fontenay-aux-Roses, France
5
Université Paris-Saclay, CEA, CNRS, UMR9199 Neurodegenerative Diseases Laboratory, 92265 Fontenay-aux-Roses, France
*
Authors to whom correspondence should be addressed.
J. Clin. Med. 2020, 9(2), 589; https://doi.org/10.3390/jcm9020589
Received: 10 January 2020 / Revised: 17 February 2020 / Accepted: 18 February 2020 / Published: 21 February 2020
(This article belongs to the Special Issue Therapies for Hearing Loss)
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. View Full-Text
Keywords: rAAV; cochlear gene therapy; clinical application; genetic deafness; routes of delivery; serotypes; targets gene addition; RNAi; gene editing rAAV; cochlear gene therapy; clinical application; genetic deafness; routes of delivery; serotypes; targets gene addition; RNAi; gene editing
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MDPI and ACS Style

Blanc, F.; Mondain, M.; Bemelmans, A.-P.; Affortit, C.; Puel, J.-L.; Wang, J. rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application. J. Clin. Med. 2020, 9, 589.

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