Article
Generation of an isogenic, gene-corrected control cell line of the spinocerebellar ataxia type 2 patient-derived iPSC line H266.
Stem cell research - 1 Jan 2016
Marthaler Adele G, Tubsuwan Alisa, Schmid Benjamin, Poulsen Ulla B, Engelbrecht Alexander F, Mau-Holzmann Ulrike A, Hyttel Poul, Nielsen Troels T, Nielsen Jørgen E, Holst Bjørn
Abstract excerpt
Spinocerebellar ataxia type 2 (SCA2) is a neurodegenerative disease primarily affecting the cerebellum. Very little is known about the molecular mechanisms underlying the disease and, to date, no cure or treatment is available. We have successfully generated bona fide induced pluripotent stem cell (iPSC) lines of SCA2 patients in order to study a disease-specific phenotype. Here, we demonstrate the gene...
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