Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice.
AAV
donor variability
gene therapy
hemophilia
human hepatocytes
humanized mice
liver
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:
11 Sep 2020
11 Sep 2020
Historique:
received:
24
01
2020
accepted:
27
05
2020
entrez:
9
7
2020
pubmed:
9
7
2020
medline:
9
7
2020
Statut:
epublish
Résumé
Adeno-associated virus (AAV) vector serotypes vary in their ability to transduce hepatocytes from different species. Chimeric mouse models harboring human hepatocytes have shown translational promise for liver-directed gene therapies. However, many variables that influence human hepatocyte transduction and transgene expression in such models remain poorly defined. Here, we aimed to test whether three experimental conditions influence AAV transgene expression in immunodeficient, fumaryl-acetoactetate-hydrolase-deficient (
Identifiants
pubmed: 32637450
doi: 10.1016/j.omtm.2020.05.033
pii: S2329-0501(20)30120-0
pmc: PMC7326722
doi:
Types de publication
Journal Article
Langues
eng
Pagination
189-198Subventions
Organisme : NIAID NIH HHS
ID : R01 AI051390
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL131093
Pays : United States
Informations de copyright
© 2020 The Authors.
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