Generation of an induced pluripotent stem cell line (XHCSUi001-A) from urine cells of a patient with spinocerebellar ataxia type 3


The induced pluripotent stem cell (iPSC) line XHCSUi001-A generated from urine cells of a female spinocerebellar ataxia type 3/Machado-Joseph disease (SCA3/MJD) patient by using the integration-free methods. The induced XHCSUi001-A retained the disease-causing ATXN3 mutation, expressed pluripotency markers, exhibited a normal karyotype and retained the ability to differentiate into the three germ layers in-vitro and in-vivo. This newly induced iPSC line could be a potential tool for researching the disease-specific mechanisms and drug screening of SCA3/MJD. Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.

Authors He L, Ye W, Chen Z, Wang C, Zhao H, Li S, Peng L, Han X, Zhou T, Li Z, Tang B, Jiang H
Journal Stem cell research
Publication Date 2019 Oct;40:101555
PubMed 31491691
DOI 10.1016/j.scr.2019.101555

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