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


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. Copyright © 2020. Published by Elsevier B.V.

Authors Ohta E, Sone T, Ukai H, Hisamatsu T, Kitagawa T, Ishikawa M, Nagai M, Ueda HR, Obata F, Okano H
Journal Stem cell research
Publication Date 2020 Dec;49:102073
PubMed 33181472
DOI 10.1016/j.scr.2020.102073

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