从患有肾上腺脑白质营养不良（AMN）的X连锁肾上腺脑白质营养不良（X-ALD）患者中生成两条诱导多能干细胞（iPSC）系。
Generation of two induced pluripotent stem cell (iPSC) lines from X-linked adrenoleukodystrophy (X-ALD) patients with adrenomyeloneuropathy (AMN).
作者信息
Son Daryeon, Quan Zhejiu, Kang Phil Jun, Park Gyuman, Kang Hoon-Chul, You Seungkwon
机构信息
Laboratory of Cell Function Regulation, Department of Biotechnology, College of Life Sciences and Biotechnology, Korea University, Seoul 136-701, Republic of Korea.
Division of Pediatric Neurology, Department of Pediatrics, Severance Children's Hospital, Epilepsy Research Institute, Seoul 03722, Republic of Korea.
出版信息
Stem Cell Res. 2017 Dec;25:46-49. doi: 10.1016/j.scr.2017.10.003. Epub 2017 Oct 12.
X-linked adrenoleukodystrophy (X-ALD) is an inherited disorder caused by a mutation in the ATP-binding cassette transporter subfamily D member 1 (ABCD1) gene. We generated two induced pluripotent stem cell (iPSC) lines from X-ALD patients with adrenomyeloneuropathy (AMN) by Sendai virus containing OCT4, SOX2, KLF4 and c-MYC. Established iPSC lines expressed various pluripotency markers, had differentiation potential of three germ layers in vitro, had normal karyotype and retained ABCD1 mutation.
X连锁肾上腺脑白质营养不良（X-ALD）是一种由ATP结合盒转运体D亚家族成员1（ABCD1）基因突变引起的遗传性疾病。我们通过含有OCT4、SOX2、KLF4和c-MYC的仙台病毒，从患有肾上腺脊髓神经病（AMN）的X-ALD患者中生成了两条诱导多能干细胞（iPSC）系。建立的iPSC系表达多种多能性标志物，在体外具有三个胚层的分化潜能，具有正常的核型并保留了ABCD1突变。
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