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
STEM CELL RESEARCH(2021)
摘要
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 vector that 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.
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关键词
Induced Pluripotent Stem Cells,Pluripotent Stem Cells,Cellular Reprogramming
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