Generation of induced pluripotent stem cell line (RCMGi008-A) from human skin fibroblasts of a cystic fibrosis patient with compound heterozygous F508del/CFTRdele2.3 mutations in CFTR gene
Summary
Skin fibroblasts obtained from a 20-year-old woman with clinically manifested and genetically proven (F508del/CFTRdele2.3) cystic fibrosis were successfully transformed into induced pluripotent stem cells (iPSCs) by using Sendai virus-based reprogramming vectors including the four Yamanaka factors, OCT3/4, SOX2, KLF4, and c-MYC. The iPSCs showed a normal karyotype, expressed pluripotency markers and exhibited the potential to differentiate into three germ layers in spontaneous differentiation assay. This iPSC line may be used for development of a personalized treatment including genome editing, disease modelling, cell differentiation and organoid formation, pharmacological investigations and drug screening. Copyright © 2022. Published by Elsevier B.V.
| Authors | Kondrateva E, Panchuk I, Demchenko A, Grigorieva O, Zheglo D, Voronina E, Erofeeva A, Tabakov V, Orlova M, Lavrov A, Smirnikhina S, Kutsev S |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2022 Aug;63:102854 |
| PubMed | 35843019 |
| DOI | 10.1016/j.scr.2022.102854 |