van der Graaf Linda M, Gardiner Sarah L, Tok Merve, Brands Tom, Boogaard Merel W, Pepers Barry A, Eussen Bert, de Klein Annelies, Aziz N Ahmad, Freund Christian, Buijsen Ronald A M, van Roon-Mom Willeke M C
Department of Human Genetics, LUMC, Leiden, The Netherlands.
Department of Neurology, LUMC, Leiden, The Netherlands.
Stem Cell Res. 2019 Aug;39:101498. doi: 10.1016/j.scr.2019.101498. Epub 2019 Jul 12.
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.
