Generation of 5 induced pluripotent stem cell lines, LUMCi007-A and B and LUMCi008-A, B and C, from 2 patients with Huntington disease

从 2 名亨廷顿病患者体内生成 5 种诱导性多能干细胞系,LUMCi007-A 和 B 以及 LUMCi008-A、B 和 C

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作者:Linda M van der Graaf, Sarah L Gardiner, Merve Tok, Tom Brands, Merel W Boogaard, Barry A Pepers, Bert Eussen, Annelies de Klein, N Ahmad Aziz, Christian Freund, Ronald A M Buijsen, Willeke M C van Roon-Mom

Abstract

Huntington disease (HD) is an autosomal dominant, neurodegenerative disease caused by a CAG repeat expansion within the coding sequence of the HTT gene, resulting in a highly toxic protein with an expanded polyglutamine stretch that forms typical protein aggregates throughout the brain. We generated human induced pluripotent stem cells (hiPSCs) from two HD patients using non-integrating Sendai virus (SeV). The hiPSCs display a normal karyotype, express all pluripotency markers, have the same CAG repeat expansion as the original fibroblasts and are able to differentiate into the three germ layers in vitro.

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