Ex Vivo/In vivo Gene Editing in Hepatocytes Using "All-in-One" CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template.
Genetic Engineering
Genetics
Techniques in Genetics
Journal
iScience
ISSN: 2589-0042
Titre abrégé: iScience
Pays: United States
ID NLM: 101724038
Informations de publication
Date de publication:
24 Jan 2020
24 Jan 2020
Historique:
received:
08
10
2018
revised:
02
10
2019
accepted:
09
12
2019
pubmed:
31
12
2019
medline:
31
12
2019
entrez:
31
12
2019
Statut:
ppublish
Résumé
Adeno-associated virus (AAV)-based vectors are considered efficient and safe gene delivery systems in gene therapy. We combined two guide RNA genes, Cas9, and a self-linearizing repair template in one vector (AIO-SL) to correct fumarylacetoacetate hydrolase (FAH) deficiency in mice. The vector genome of 5.73 kb was packaged into VP2-depleted AAV particles (AAV2/8
Identifiants
pubmed: 31887661
pii: S2589-0042(19)30509-7
doi: 10.1016/j.isci.2019.100764
pmc: PMC6941859
pii:
doi:
Types de publication
Journal Article
Langues
eng
Pagination
100764Informations de copyright
Copyright © 2019 The Author(s). Published by Elsevier Inc. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of Interests The authors declare no competing interests.
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