Generation and characterization of induced pluripotent stem cell lines derived from skin fibroblasts of patients with adrenoleukodystrophy
Summary
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. Copyright © 2023 The Authors. Published by Elsevier B.V. All rights reserved.
| Authors | Wang QH, Liu LY, Wang YY, He W, Wang J, Wang J, Zou LP |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2023 Dec;73:103243 |
| PubMed | 37948838 |
| DOI | 10.1016/j.scr.2023.103243 |