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

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

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...

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Bibliographic Details
Main Authors: Fabian Blanc, Michel Mondain, Alexis-Pierre Bemelmans, Corentin Affortit, Jean-Luc Puel, Jing Wang
Format: Article
Language:English
Published: MDPI AG 2020-02-01
Series:Journal of Clinical Medicine
Subjects:
raav
cochlear gene therapy
clinical application
genetic deafness
routes of delivery
serotypes
targets gene addition
rnai
gene editing
Online Access:https://www.mdpi.com/2077-0383/9/2/589

Internet

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

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