TALEN mediated gene editing in a mouse model of Fanconi anemia.
Animals
Blotting, Western
Chromosomal Instability
/ genetics
Fanconi Anemia
/ genetics
Female
Flow Cytometry
Gene Editing
/ methods
HEK293 Cells
Hematopoietic Stem Cells
/ cytology
Humans
In Situ Hybridization, Fluorescence
Mice, Inbred C57BL
Pregnancy
Transcription Activator-Like Effector Nucleases
/ genetics
Journal
Scientific reports
ISSN: 2045-2322
Titre abrégé: Sci Rep
Pays: England
ID NLM: 101563288
Informations de publication
Date de publication:
24 04 2020
24 04 2020
Historique:
received:
30
05
2019
accepted:
28
02
2020
entrez:
26
4
2020
pubmed:
26
4
2020
medline:
22
12
2020
Statut:
epublish
Résumé
The promising ability to genetically modify hematopoietic stem and progenitor cells by precise gene editing remains challenging due to their sensitivity to in vitro manipulations and poor efficiencies of homologous recombination. This study represents the first evidence of implementing a gene editing strategy in a murine safe harbor locus site that phenotypically corrects primary cells from a mouse model of Fanconi anemia A. By means of the co-delivery of transcription activator-like effector nucleases and a donor therapeutic FANCA template to the Mbs85 locus, we achieved efficient gene targeting (23%) in mFA-A fibroblasts. This resulted in the phenotypic correction of these cells, as revealed by the reduced sensitivity of these cells to mitomycin C. Moreover, robust evidence of targeted integration was observed in murine wild type and FA-A hematopoietic progenitor cells, reaching mean targeted integration values of 21% and 16% respectively, that were associated with the phenotypic correction of these cells. Overall, our results demonstrate the feasibility of implementing a therapeutic targeted integration strategy into the mMbs85 locus, ortholog to the well-validated hAAVS1, constituting the first study of gene editing in mHSC with TALEN, that sets the basis for the use of a new safe harbor locus in mice.
Identifiants
pubmed: 32332829
doi: 10.1038/s41598-020-63971-z
pii: 10.1038/s41598-020-63971-z
pmc: PMC7181878
doi:
Substances chimiques
Transcription Activator-Like Effector Nucleases
EC 3.1.-
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
6997Références
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pubmed: 21903629
pmcid: 21903629