Journal
STEM CELL RESEARCH
Volume 29, Issue -, Pages 125-128Publisher
ELSEVIER
DOI: 10.1016/j.scr.2018.03.018
Keywords
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Funding
- Dutch SCA1 Families Fund
- Dutch Brain Foundation (Nederlandse Hersenstichting) [HA2016-02-02]
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Spinocerebellar ataxia type 1 (SCA1) is a hereditary neurodegenerative disease caused by a CAG repeat expansion in exon 8 of the ATXN1 gene. We generated induced pluripotent stem cells (hiPSCs) from a SCA1 patient and his non-affected sister by using non-integrating Sendai Viruses (SeV). The resulting hiPSCs are SeVfree, express pluripotency markers, display a normal karyotype, retain the mutation (length of the CAG repeat expansion in the ATXN1 gene) and are able to differentiate into the three germ layers in vitro. (C) 2018 The Authors. Published by Elsevier B.V.
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