Generation of induced pluripotent stem cell line (RCMGi009-A) from urine cells of patient with fibrodysplasia ossificans progressiva


Urine cells obtained from a 14-year-old man with genetically proven (ACVR1: c.6176G > A) and clinically manifested fibrodysplasia ossificans progressiva were successfully transformed into induced pluripotent stem cells by using Sendai virus-based reprogramming vectors including the four Yamanaka factors such as OCT3/4, SOX2, KLF4, and c-MYC. These iPSCs expressed pluripotency markers, exhibited the potential to differentiate into three germ layers in spontaneous differentiation assay and had a normal karyotype. The iPSC line may provide a model for development of a personalized treatment including genome editing and drug screening, may be used for disease modelling, cell differentiation and pharmacological investigations. . Copyright © 2023 The Author(s). Published by Elsevier B.V. All rights reserved.

Authors Kondrateva E, Grigorieva O, Kurshakova E, Panchuk I, Pozhitnova V, Voronina E, Tabakov V, Orlova M, Lavrov A, Smirnikhina S, Kutsev S
Journal Stem cell research
Publication Date 2023 Aug;70:103133
PubMed 37307755
DOI 10.1016/j.scr.2023.103133

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