Generation of 3 spinocerebellar ataxia type 1 (SCA1) patient-derived induced pluripotent stem cell lines LUMCi002-A, B, and C and 2 unaffected sibling control induced pluripotent stem cell lines LUMCi003-A and B
oleh: Ronald A.M. Buijsen, Sarah L. Gardiner, Marga J. Bouma, Linda M. van der Graaf, Merel W. Boogaard, Barry A. Pepers, Bert Eussen, Annelies de Klein, Christian Freund, Willeke M.C. van Roon-Mom
| Format: | Article |
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| Diterbitkan: | Elsevier 2018-05-01 |
Deskripsi
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.