抄録
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.
本文言語 | 英語 |
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論文番号 | 102073 |
ジャーナル | Stem Cell Research |
巻 | 49 |
DOI | |
出版ステータス | 出版済み - 12-2020 |
外部発表 | はい |
All Science Journal Classification (ASJC) codes
- 発生生物学
- 細胞生物学