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

Etsuro Ohta, Takefumi Sone, Hideki Ukai, Tomoko Hisamatsu, Tokiko Kitagawa, Mitsuru Ishikawa, Makiko Nagai, Hiroki R. Ueda, Fumiya Obata, Hideyuki Okano

研究成果: ジャーナルへの寄稿学術論文査読

4 被引用数 (Scopus)

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
論文番号102073
ジャーナルStem Cell Research
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

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