Substantial restoration of night vision in adult mice with congenital stationary night blindness.
AAV
CSNB
LRIT3
bipolar cells
congenital stationary night blindness
gene therapy
photoreceptors
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
10 Sep 2021
10 Sep 2021
Historique:
received:
26
08
2020
accepted:
13
05
2021
entrez:
17
8
2021
pubmed:
18
8
2021
medline:
18
8
2021
Statut:
epublish
Résumé
Complete congenital stationary night blindness (cCSNB) due to mutations in
Identifiants
pubmed: 34401402
doi: 10.1016/j.omtm.2021.05.008
pii: S2329-0501(21)00092-9
pmc: PMC8339357
doi:
Types de publication
Journal Article
Langues
eng
Pagination
15-25Informations de copyright
© 2021 The Authors.
Déclaration de conflit d'intérêts
The funders had no role in study design, data collection, analysis and interpretation, decision to publish, or preparation of the manuscript. J.V., D.D., S.P., I.A., J.-A.S., and C.Z. are inventors on a pending patent application on “Treatment of congenital stationary night blindness using gene therapy” (SL0160 [CLBEnaco-F2478 36WO]). D.D. is an inventor on a patent of adeno-associated virus virions with variant capsid and methods of use thereof with royalties paid to Avalanche Biotech (WO2012145601 A2).
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