Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome.
CRISPR/Cas9
Netherton syndrome
disease modeling
gene editing
genodermatosis
human keratinocytes
skin equivalents.
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:
07
02
2020
accepted:
27
05
2020
entrez:
9
7
2020
pubmed:
9
7
2020
medline:
9
7
2020
Statut:
epublish
Résumé
Current efforts to find specific genodermatoses treatments and define precise pathogenesis mechanisms require appropriate surrogate models with human cells. Although transgenic and gene knockout mouse models for several of these disorders exist, they often fail to faithfully replicate the clinical and histopathological features of the human skin condition. We have established a highly efficient method for precise deletion of critical gene sequences in primary human keratinocytes, based on CRISPR-Cas9-mediated gene editing. Using this methodology, in the present study we generated a model of Netherton syndrome by disruption of
Identifiants
pubmed: 32637457
doi: 10.1016/j.omtm.2020.05.031
pii: S2329-0501(20)30118-2
pmc: PMC7329935
doi:
Types de publication
Journal Article
Langues
eng
Pagination
280-290Informations de copyright
© 2020 The Author(s).
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