Generation and characterization of induced pluripotent stem cell lines derived from skin fibroblasts of patients with adrenoleukodystrophy

Autor: Qiu-Hong Wang, Li-Ying Liu, Yang-Yang Wang, Wen He, Jia Wang, Jing Wang, Li-Ping Zou
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: Stem Cell Research, Vol 73, Iss , Pp 103243- (2023)
Druh dokumentu: article
ISSN: 1873-5061
DOI: 10.1016/j.scr.2023.103243
Popis: X-linked adrenoleukodystrophy (ALD) is a rare peroxisome disease with phenotypic heterogeneity. There is a lack of suitable in vitro models to study its pathogenesis. We established two strains of iPSCs from skin fibroblasts of patients with childhood cerebral ALD and Addison’s disease, respectively. CytoTune™2.0 Sendai reprogramming kit was used. The iPSC lines showed typical stem cell morphology, normal karyotype, and carrying ABCD1 variation. The iPSC lines express pluripotency markers, and have the capacity to differentiate into three germ layers. iPSCs can be used as an alternative cell source for ALD in vitro model to study its pathogenesis and therapeutic strategies.
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