Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

Mutations in the mechanotransduction channel component TMC1/2 cause deafness. Here, the authors use a synthetic AAV to replace TMC1 and 2 in the inner ear and show restoration of cochlear and vestibular function, of neuronal reponses in the auditory cortex and of hearing and balance in mice.

Bibliographic Details
Main Authors: Carl A. Nist-Lund, Bifeng Pan, Amy Patterson, Yukako Asai, Tianwen Chen, Wu Zhou, Hong Zhu, Sandra Romero, Jennifer Resnik, Daniel B. Polley, Gwenaelle S. Géléoc, Jeffrey R. Holt
Format: Article
Language:English
Published: Nature Publishing Group 2019-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-018-08264-w
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spelling doaj-998ee4b6a33b41c587d88c8b399215ea2021-05-11T12:10:16ZengNature Publishing GroupNature Communications2041-17232019-01-0110111410.1038/s41467-018-08264-wImproved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disordersCarl A. Nist-Lund0Bifeng Pan1Amy Patterson2Yukako Asai3Tianwen Chen4Wu Zhou5Hong Zhu6Sandra Romero7Jennifer Resnik8Daniel B. Polley9Gwenaelle S. Géléoc10Jeffrey R. Holt11Department of Otolaryngology and F.M. Kirby Neurobiology Center, Boston Children’s HospitalDepartment of Otolaryngology and F.M. Kirby Neurobiology Center, Boston Children’s HospitalDepartment of Otolaryngology and F.M. Kirby Neurobiology Center, Boston Children’s HospitalDepartment of Otolaryngology and F.M. Kirby Neurobiology Center, Boston Children’s HospitalDepartment of Otolaryngology and Communicative Sciences, University of Mississippi Medical CenterDepartment of Otolaryngology and Communicative Sciences, University of Mississippi Medical CenterDepartment of Otolaryngology and Communicative Sciences, University of Mississippi Medical CenterEaton-Peabody Laboratories, Massachusetts Eye and Ear InfirmaryDepartment of Otolaryngology, Harvard Medical SchoolDepartment of Otolaryngology, Harvard Medical SchoolDepartment of Otolaryngology and F.M. Kirby Neurobiology Center, Boston Children’s HospitalDepartment of Otolaryngology and F.M. Kirby Neurobiology Center, Boston Children’s HospitalMutations in the mechanotransduction channel component TMC1/2 cause deafness. Here, the authors use a synthetic AAV to replace TMC1 and 2 in the inner ear and show restoration of cochlear and vestibular function, of neuronal reponses in the auditory cortex and of hearing and balance in mice.https://doi.org/10.1038/s41467-018-08264-w
collection DOAJ
language English
format Article
sources DOAJ
author Carl A. Nist-Lund
Bifeng Pan
Amy Patterson
Yukako Asai
Tianwen Chen
Wu Zhou
Hong Zhu
Sandra Romero
Jennifer Resnik
Daniel B. Polley
Gwenaelle S. Géléoc
Jeffrey R. Holt
spellingShingle Carl A. Nist-Lund
Bifeng Pan
Amy Patterson
Yukako Asai
Tianwen Chen
Wu Zhou
Hong Zhu
Sandra Romero
Jennifer Resnik
Daniel B. Polley
Gwenaelle S. Géléoc
Jeffrey R. Holt
Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
Nature Communications
author_facet Carl A. Nist-Lund
Bifeng Pan
Amy Patterson
Yukako Asai
Tianwen Chen
Wu Zhou
Hong Zhu
Sandra Romero
Jennifer Resnik
Daniel B. Polley
Gwenaelle S. Géléoc
Jeffrey R. Holt
author_sort Carl A. Nist-Lund
title Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
title_short Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
title_full Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
title_fullStr Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
title_full_unstemmed Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
title_sort improved tmc1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
publisher Nature Publishing Group
series Nature Communications
issn 2041-1723
publishDate 2019-01-01
description Mutations in the mechanotransduction channel component TMC1/2 cause deafness. Here, the authors use a synthetic AAV to replace TMC1 and 2 in the inner ear and show restoration of cochlear and vestibular function, of neuronal reponses in the auditory cortex and of hearing and balance in mice.
url https://doi.org/10.1038/s41467-018-08264-w
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