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
Summary
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. Copyright © 2021 The Author(s). Published by Elsevier B.V. All rights reserved.
| Authors | Kondrateva E, Demchenko A, Slesarenko Y, Pozhitnova V, Yasinovsky M, Amelina E, Tabakov V, Voronina E, Lavrov A, Smirnikhina S |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2021 Apr;52:102232 |
| PubMed | 33607467 |
| DOI | 10.1016/j.scr.2021.102232 |