Generation of an induced pluripotent stem cell (iPSC) line from a 42-year-old adult cerebral type X-linked adrenoleukodystrophy (X-ALD) patient

Gyu-Bum Yeon; Daryeon Son; Phil Jun Kang; Seung-Min Oh; Hun Lim; Hoon-Chul Kang; Seungkwon You; Dae-Sung Kim

doi:10.1016/j.scr.2019.101425

Generation of an induced pluripotent stem cell (iPSC) line from a 42-year-old adult cerebral type X-linked adrenoleukodystrophy (X-ALD) patient

Stem Cell Res. 2019 Apr:36:101425. doi: 10.1016/j.scr.2019.101425. Epub 2019 Mar 21.

Authors

Gyu-Bum Yeon¹, Daryeon Son¹, Phil Jun Kang¹, Seung-Min Oh¹, Hun Lim¹, Hoon-Chul Kang², Seungkwon You³, Dae-Sung Kim⁴

Affiliations

¹ Department of Biotechnology, College of Life Science and Biotechnology, Korea University, 145 Anam-ro, Seongbuk-gu, Seoul 02841, Republic of Korea.
² Division of Pediatric Neurology, Department of Pediatrics, Severance Children's Hospital, Yonsei University College of Medicine, 50-1 Yonsei-ro, Seodaemun-gu, Seoul 03722, Republic of Korea.
³ Department of Biotechnology, College of Life Science and Biotechnology, Korea University, 145 Anam-ro, Seongbuk-gu, Seoul 02841, Republic of Korea. Electronic address: bioseung@korea.ac.kr.
⁴ Department of Biotechnology, College of Life Science and Biotechnology, Korea University, 145 Anam-ro, Seongbuk-gu, Seoul 02841, Republic of Korea; Department of Pediatrics, Korea University College of Medicine, Guro Hospital, 97 Gurodong-gil, Guro-gu, Seoul 08308, Republic of Korea. Electronic address: sonnet10@korea.ac.kr.

PMID: 30921588
DOI: 10.1016/j.scr.2019.101425

Abstract

X-linked Adrenoleukodystrophy (X-ALD) is a neuro-metabolic disorder that is caused by malfunction of a peroxisomal transporter protein, adenosine ATP-binding cassette transporter superfamily D member 1 (ABCD1). We established an induced pluripotent stem cell (iPSC) line from a 42-year-old male X-ALD patient-derived dermal fibroblasts with Sendai virus-mediated reprogramming. Established iPSCs stably expanded, expressed genes of pluripotency, and maintained normal karyotype. In vitro differentiation assay revealed the characteristics of all three germ layers.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

ATP Binding Cassette Transporter, Subfamily D, Member 1 / genetics*
Adrenoleukodystrophy / genetics*
Adult
Cell Differentiation
Cell Line*
Cellular Reprogramming Techniques
Humans
INDEL Mutation
Induced Pluripotent Stem Cells*
Karyotype
Male

Substances

ABCD1 protein, human
ATP Binding Cassette Transporter, Subfamily D, Member 1