Overloaded Adeno-Associated Virus as a Novel Gene Therapeutic Tool for Otoferlin-Related Deafness.

AAV (adeno-associated virus) Auditory Neuroscience deafness/hearing loss gene therapy and therapeutic delivery in vivo organotypic culture model otoferlin preclinical

Journal

Frontiers in molecular neuroscience
ISSN: 1662-5099
Titre abrégé: Front Mol Neurosci
Pays: Switzerland
ID NLM: 101477914

Informations de publication

Date de publication:
2020
Historique:
received: 28 08 2020
accepted: 04 12 2020
entrez: 25 1 2021
pubmed: 26 1 2021
medline: 26 1 2021
Statut: epublish

Résumé

Hearing impairment is the most common sensory disorder in humans. So far, rehabilitation of profoundly deaf subjects relies on direct stimulation of the auditory nerve through cochlear implants. However, in some forms of genetic hearing impairment, the organ of Corti is structurally intact and therapeutic replacement of the mutated gene could potentially restore near natural hearing. In the case of defects of the otoferlin gene (

Identifiants

pubmed: 33488357
doi: 10.3389/fnmol.2020.600051
pmc: PMC7817888
doi:

Types de publication

Journal Article

Langues

eng

Pagination

600051

Informations de copyright

Copyright © 2021 Rankovic, Vogl, Dörje, Bahader, Duque-Afonso, Thirumalai, Weber, Kusch, Strenzke and Moser.

Déclaration de conflit d'intérêts

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Références

J Neurosci. 2019 Apr 17;39(16):3159-3169
pubmed: 30755487
Nat Protoc. 2006;1(3):1412-28
pubmed: 17406430
Annu Rev Neurosci. 2019 Jul 8;42:67-86
pubmed: 30699050
Mol Ther Methods Clin Dev. 2015 Mar 25;2:15009
pubmed: 26029720
Hear Res. 2020 Sep 1;394:107882
pubmed: 31980281
Nat Biotechnol. 2016 Feb;34(2):204-9
pubmed: 26829320
Nat Neurosci. 2010 Jul;13(7):869-76
pubmed: 20562868
Science. 1970 Mar 13;167(3924):1517-8
pubmed: 5415287
J Neurosci. 2010 Jun 2;30(22):7587-97
pubmed: 20519533
Nat Methods. 2012 Jul;9(7):671-5
pubmed: 22930834
Nat Rev Neurol. 2016 Mar;12(3):135-49
pubmed: 26891769
J Neurosci. 2019 May 1;39(18):3394-3411
pubmed: 30833506
EMBO J. 2015 Nov 3;34(21):2686-702
pubmed: 26446278
Cell. 2016 Jun 16;165(7):1789-1802
pubmed: 27238021
Mol Ther. 2018 Feb 7;26(2):524-541
pubmed: 29292161
J Med Genet. 2006 Jul;43(7):576-81
pubmed: 16371502
J Virol. 1998 Nov;72(11):8568-77
pubmed: 9765395
Nature. 2017 Oct 12;550(7676):314
pubmed: 29052639
Mol Ther. 2001 Oct;4(4):383-91
pubmed: 11592843
Physiol Rev. 2020 Oct 1;100(4):1467-1525
pubmed: 32191560
EMBO J. 2016 Dec 1;35(23):2536-2552
pubmed: 27458190
Elife. 2017 Nov 07;6:
pubmed: 29111973
Mol Ther. 2017 Feb 1;25(2):379-391
pubmed: 28082074
Cell. 2006 Oct 20;127(2):277-89
pubmed: 17055430
EMBO J. 2019 Mar 1;38(5):
pubmed: 30733243
J Cell Biol. 2010 Oct 4;191(1):187-97
pubmed: 20921140
J Virol. 2005 Aug;79(15):9933-44
pubmed: 16014954
Mol Ther. 2017 May 3;25(5):1076-1094
pubmed: 28391961
Anat Rec (Hoboken). 2012 Nov;295(11):1812-29
pubmed: 23044516
Nat Biotechnol. 2017 Mar;35(3):264-272
pubmed: 28165476
Neuron. 2012 Jul 26;75(2):283-93
pubmed: 22841313
PLoS One. 2014 Mar 14;9(3):e91676
pubmed: 24632841
N Engl J Med. 2000 Apr 13;342(15):1101-9
pubmed: 10760311
Hear Res. 2015 Dec;330(Pt B):200-12
pubmed: 26188103
EMBO Mol Med. 2019 Jan;11(1):
pubmed: 30509897
EMBO J. 2016 Dec 1;35(23):2519-2535
pubmed: 27729456
J Cell Sci. 2015 Feb 15;128(4):638-44
pubmed: 25609709
J Clin Invest. 2018 Aug 1;128(8):3382-3401
pubmed: 29985171
Nat Genet. 1999 Apr;21(4):363-9
pubmed: 10192385
Proc Natl Acad Sci U S A. 2019 Mar 5;116(10):4496-4501
pubmed: 30782832
Nat Commun. 2017 Oct 12;8(1):886
pubmed: 29026089
Sci Transl Med. 2015 Jul 8;7(295):295ra108
pubmed: 26157030
Mol Ther. 2013 Dec;21(12):2205-16
pubmed: 23939025
Mol Ther. 2014 Mar;22(3):567-574
pubmed: 23941813
Neuroscience. 2007 Nov 9;149(3):673-84
pubmed: 17869440
Gene Ther. 1999 Jun;6(6):973-85
pubmed: 10455399
J Clin Invest. 2008 May;118(5):1955-64
pubmed: 18414684
PLoS One. 2012;7(6):e39233
pubmed: 22720081
Hum Mutat. 2008 Jun;29(6):823-31
pubmed: 18381613
Hear Res. 2005 Apr;202(1-2):63-73
pubmed: 15811700
Physiol Rev. 2020 Jan 1;100(1):103-144
pubmed: 31373863

Auteurs

Vladan Rankovic (V)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Restorative Cochlear Genomics Group, Auditory Neuroscience and Optogenetics Laboratory, German Primate Center, Göttingen, Germany.

Christian Vogl (C)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Presynaptogenesis and Intracellular Transport in Hair Cells Group, Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Collaborative Research Center 889, University of Göttingen, Göttingen, Germany.

Nele M Dörje (NM)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Presynaptogenesis and Intracellular Transport in Hair Cells Group, Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.

Iman Bahader (I)

Collaborative Research Center 889, University of Göttingen, Göttingen, Germany.
Auditory Systems Physiology Group, Institute for Auditory Neuroscience and Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany.

Carlos J Duque-Afonso (CJ)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Auditory Neuroscience Group, Max Planck Institute of Experimental Medicine, Göttingen, Germany.
Multiscale Bioimaging Cluster of Excellence (MBExC), University of Göttingen, Göttingen, Germany.

Anupriya Thirumalai (A)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.

Thomas Weber (T)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.

Kathrin Kusch (K)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Restorative Cochlear Genomics Group, Auditory Neuroscience and Optogenetics Laboratory, German Primate Center, Göttingen, Germany.

Nicola Strenzke (N)

Collaborative Research Center 889, University of Göttingen, Göttingen, Germany.
Auditory Systems Physiology Group, Institute for Auditory Neuroscience and Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany.

Tobias Moser (T)

Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
Collaborative Research Center 889, University of Göttingen, Göttingen, Germany.
Auditory Neuroscience Group, Max Planck Institute of Experimental Medicine, Göttingen, Germany.
Multiscale Bioimaging Cluster of Excellence (MBExC), University of Göttingen, Göttingen, Germany.
Synaptic Nanophysiology Group, Max Planck Institute of Biophysical Chemistry, Göttingen, Germany.
Auditory Neuroscience and Optogenetics Laboratory, German Primate Center, Göttingen, Germany.

Classifications MeSH