Neonatal AAV gene therapy rescues hearing in a mouse model of SYNE4 deafness.
DFNB76
Nesprin-4
SYNE4
deafness
gene therapy
Journal
EMBO molecular medicine
ISSN: 1757-4684
Titre abrégé: EMBO Mol Med
Pays: England
ID NLM: 101487380
Informations de publication
Date de publication:
05 02 2021
05 02 2021
Historique:
received:
08
08
2020
revised:
10
11
2020
accepted:
18
11
2020
pubmed:
23
12
2020
medline:
26
10
2021
entrez:
22
12
2020
Statut:
ppublish
Résumé
Genetic variants account for approximately half the cases of congenital and early-onset deafness. Methods and technologies for viral delivery of genes into the inner ear have evolved over the past decade to render gene therapy a viable and attractive approach for treatment. Variants in SYNE4, encoding the protein nesprin-4, a member of the linker of nucleoskeleton and cytoskeleton (LINC), lead to DFNB76 human deafness. Syne4
Identifiants
pubmed: 33350593
doi: 10.15252/emmm.202013259
pmc: PMC7863404
doi:
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e13259Subventions
Organisme : NICHD NIH HHS
ID : P50 HD105351
Pays : United States
Informations de copyright
© 2020 The Authors. Published under the terms of the CC BY 4.0 license.
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