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 pheno...

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Veröffentlicht in:Stem cell research 2020-12, Vol.49, p.102073-102073, Article 102073
Hauptverfasser: Ohta, Etsuro, Sone, Takefumi, Ukai, Hideki, Hisamatsu, Tomoko, Kitagawa, Tokiko, Ishikawa, Mitsuru, Nagai, Makiko, Ueda, Hiroki R., Obata, Fumiya, Okano, Hideyuki
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Sprache:eng
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Zusammenfassung: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.
ISSN:1873-5061
1876-7753
DOI:10.1016/j.scr.2020.102073