Generation of induced pluripotent stem cell line (RCMGi001-A) from human skin fibroblasts of a cystic fibrosis patient with p.F508del mutation
Ekaterina Kondrateva, Elmira P. Adilgereeva, E. Amelina, Vyacheslav Tabakov, Anna Demchenko, Kirill Ustinov, Matvey Yasinovsky, Е. С. Воронина, А. В. Лавров, Svetlana Smirnikhina
Abstract
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.