Generation of human iPS cell line IBCHi002-A from spinocerebellar ataxia type 3/Machado-Joseph disease patient's fibroblasts


Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD), is autosomal-dominant neurodegenerative disease caused by an expansion of polyglutamine-encoding CAG repeats in the ATXN3 gene. Here we established IBCHi002-A induced pluripotent stem cells (iPSCs) line generated from SCA3 patient fibroblasts by using non-integrative Sendai-virus delivery system of four reprogramming factors. This cellular model provides a valid platform for study SCA3 pathogenesis and potential therapies for this so far incurable disease. Copyright © 2020 The Author(s). Published by Elsevier B.V. All rights reserved.

Authors Ciolak A, Krzyzosiak WJ, Kozlowska E, Fiszer A
Journal Stem cell research
Publication Date 2020 May;45:101796
PubMed 32361312
DOI 10.1016/j.scr.2020.101796

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