Generation of induced pluripotent stem cell line (RCMGi001-A) from human skin fibroblasts of a cystic fibrosis patient with p.F508del mutation


Skin fibroblasts obtained from a 27-year-old man with clinically manifested and genetically proven (F508del/F508del) 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 subsequently used for development of a personalized etiotropic treatment including genome editing, and for disease modelling and drug screening. Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.

Authors Kondrateva E, Adilgereeva E, Amelina E, Tabakov V, Demchenko A, Ustinov K, Yasinovsky M, Voronina E, Lavrov A, Smirnikhina S
Journal Stem cell research
Publication Date 2020 Oct;48:101933
PubMed 32777768
DOI 10.1016/j.scr.2020.101933

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