SCIENTIFIC LITERATURE · MED

Generation of gene-corrected iPSCs line (KEIUi001-A) from a PARK8 patient iPSCs with familial Parkinson's disease carrying the I2020T mutation in LRRK2.

Ohta E, Sone T, Ukai H, Hisamatsu T, Kitagawa T, Ishikawa M, Nagai M, Ueda HR, Obata F, Okano H.

Stem Cell Res · 2020-11-03

DOI: 10.1016/j.scr.2020.102073PMID: 33181472

Check source for accessResearch Support, Non-U.S. Gov't; Journal ArticleOriginal source ↗

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Leucine-rich repeat kinase 2 (LRRK2) is the causal gene of the autosomal dominant hereditary form of Parkinson's disease (PD), PARK8. We have previously reported that induced pluripotent stem cells (iPSCs) from a PARK8 patient with I2020T LRRK2 mutation replicated to some extent the pathologic phenotype evident in the brain of PD patients. In the present study, we generated gene-corrected iPSCs line, KEIUi001-A, using TALEN-mediated genome editing. KEIUi001-A retained a normal karyotype and pluripotency, i.e. the capacity to differentiate into cell types of the three germ layers. This iPSCs will be valuable for clarifying various aspects of LRRK2-related pathology.

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