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