Generation of an induced pluripotent stem cell line (KCGMHi003-A) from a patient with spinocerebellar ataxia type 8 (SCA8)
Summary
Spinocerebellar Ataxia Type 8 (SCA8) is a rare, dominantly inherited neurodegenerative disorder characterized by progressive ataxia and nystagmus, and dysarthria. SCA8 is caused by bidirectional CTG/CAG repeat expansion in the ATXN8OS and ATXN8 gene. Peripheral blood mononuclear cells obtained from a SCA8 patient were successfully transformed into induced pluripotent stem cells (iPSC) (KCGMHi003-A) using Sendai virus. Our approach provided a resource for future pathogenesis study and drug screening of SCA8. Copyright © 2025 The Author(s). Published by Elsevier B.V. All rights reserved.
| Authors | Lan MY, Lim SJ, Lu HE, Tsai MH |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2025 Dec 5;90:103879 |
| PubMed | 41353794 |
| DOI | 10.1016/j.scr.2025.103879 |