Generation of two induced pluripotent stem cell lines (RCMGi004-A and -B) from human skin fibroblasts of a cystic fibrosis patient with compound heterozygous F508del/W1282X mutations in CFTR gene.
Journal
Stem cell research
ISSN: 1876-7753
Titre abrégé: Stem Cell Res
Pays: England
ID NLM: 101316957
Informations de publication
Date de publication:
04 2021
04 2021
Historique:
received:
22
09
2020
revised:
22
01
2021
accepted:
04
02
2021
pubmed:
20
2
2021
medline:
1
7
2021
entrez:
19
2
2021
Statut:
ppublish
Résumé
Skin fibroblasts obtained from a 28-year-old man with clinically manifested and genetically proven (F508del/W1282X) cystic fibrosis were successfully transformed into induced pluripotent stem cells (iPSCs) by using non-viral, non-integrating, self-replicating RNA reprogramming vectorthat contains five reprogramming factors: OCT4, KLF4, SOX2, GLIS1, and c-MYC as well as a puromycin-resistance gene. Two iPSC lines showed a normal karyotype, expressed pluripotency markers and exhibited the potential to differentiate into three germ layers in spontaneous differentiation assay. These iPSC lines may be subsequently used for development of a personalized etiotropic treatment,disease modelling, cell differentiation and organoid formation, pharmacological investigations and drug screening.
Identifiants
pubmed: 33607467
pii: S1873-5061(21)00078-7
doi: 10.1016/j.scr.2021.102232
pii:
doi:
Substances chimiques
CFTR protein, human
0
KLF4 protein, human
0
Kruppel-Like Factor 4
0
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
Types de publication
Case Reports
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
102232Informations de copyright
Copyright © 2021 The Author(s). Published by Elsevier B.V. All rights reserved.