Generation of a human induced pluripotent stem cell line PUMCi001-A from a patient with Krabbe disease

Wei Wang; Ya-feng Lv; Yan-jun Zhang; Wen-ji Dong; Ye Zhang

Stem Cell Research (Oct 2020)

Generation of a human induced pluripotent stem cell line PUMCi001-A from a patient with Krabbe disease

Wei Wang,
Ya-feng Lv,
Yan-jun Zhang,
Wen-ji Dong,
Ye Zhang

Affiliations

Wei Wang: Department of Neurology, China-Japan Friendship Hospital, Beijing 100029 China
Ya-feng Lv: State Key Laboratory of Medical Molecular Biology, Department of Biochemistry and Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100005, China
Yan-jun Zhang: State Key Laboratory of Medical Molecular Biology, Department of Biochemistry and Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100005, China; Genmedicn Biopharma, Beijing 100176 China
Wen-ji Dong: Genmedicn Biopharma, Beijing 100176 China; Corresponding authors.
Ye Zhang: State Key Laboratory of Medical Molecular Biology, Department of Biochemistry and Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100005, China; Corresponding authors.

Journal volume & issue: Vol. 48
p. 101937

Abstract

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We have generated PUMCi001-A, an induced pluripotent stem cells (iPSC) line from dermal fibroblasts of a 13-year-old male Krabbe disease patient with two hemizygous (461C > A and 1244G > A) mutations in Galactocerebrosidase (GALC) gene using a Sendai viral delivery of OCT4, SOX2, KLF4, and c-MYC. The PUMCi001-A iPSC line carried the GALC mutations, displayed typical iPSC morphology, expressed pluripotent stem cell makers, exhibited a normal karyotype and differentiation capacity into three germ layers.

Published in Stem Cell Research

ISSN: 1873-5061 (Print); 1876-7753 (Online)
Publisher: Elsevier
Country of publisher: Netherlands
LCC subjects: Science: Biology (General)
Website: https://www.journals.elsevier.com/stem-cell-research

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