CRISPR/Cas9
gene editing
gene therapy
leukodystrophy
vanishing white matter
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:
09 Jun 2022
09 Jun 2022
Historique:
received:
27
08
2021
accepted:
19
02
2022
entrez:
23
3
2022
pubmed:
24
3
2022
medline:
24
3
2022
Statut:
epublish
Résumé
Vanishing white matter (VWM) is a leukodystrophy caused by recessive variants in subunits of eIF2B. At present, no curative treatment is available and patients often die at young age. Due to its monogenic nature, VWM is a promising candidate for the development of CRISPR/Cas9-mediated gene therapy. Here we tested a dual-AAV approach in VWM mice encoding CRISPR/Cas9 and a DNA donor template to correct a pathogenic variant in
Identifiants
pubmed: 35317047
doi: 10.1016/j.omtm.2022.02.006
pii: S2329-0501(22)00022-5
pmc: PMC8917273
doi:
Types de publication
Journal Article
Langues
eng
Pagination
17-25Informations de copyright
© 2022 The Author(s).
Déclaration de conflit d'intérêts
The authors declare no competing interests.
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