Generation of two induced pluripotent stem cell (iPSC) lines from p.F508del Cystic Fibrosis patients

从 p.F508del 囊性纤维化患者中生成两种诱导性多能干细胞 (iPSC) 系

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作者:Aarne Fleischer, Iván M Lorenzo, Esther Palomino, Trond Aasen, Fernando Gómez, Miguel Servera, Víctor J Asensio, Víctor Gálvez, Juan Carlos Izpisúa-Belmonte, Daniel Bachiller

Abstract

Cystic Fibrosis (CF) is a monogenic, lethal disease caused by mutations in the cystic fibrosis transmembrane conductance (CFTR) gene. Here we report the production of CF-iPS cell lines from two different p.F508del homozygous female patients (Table 1). Two different primary cell types, skin fibroblasts and keratinocytes, were transfected with retroviral cocktails containing four: c-MYC, KLF4, OCT4 and SOX2 (MKOS) or three: KLF4, OCT4 and SOX2 (KOS) reprogramming factors. Two fibroblast-derived MKOS lines are described in the main text. The lines carry the p.F508del mutation, have a normal karyotype, express pluripotency markers and are able to differentiate into the three germ layers.

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