TY - JOUR
T1 - Generation of gene-corrected iPSCs line (KEIUi001-A) from a PARK8 patient iPSCs with familial Parkinson's disease carrying the I2020T mutation in LRRK2
AU - Ohta, Etsuro
AU - Sone, Takefumi
AU - Ukai, Hideki
AU - Hisamatsu, Tomoko
AU - Kitagawa, Tokiko
AU - Ishikawa, Mitsuru
AU - Nagai, Makiko
AU - Ueda, Hiroki R.
AU - Obata, Fumiya
AU - Okano, Hideyuki
N1 - Funding Information:
This work was supported by Regenerative Medicine (the Program for Intractable Disease Research Utilizing Disease-specific iPS Cells and the Acceleration Program for Intractable Diseases Research Utilizing Disease-specific iPS Cells) and Practical Research Project for Rare/Intractable Diseases (H.O.) (16ek0109013h0003, 16ek0109158h0002, 16bm0609003h0005, 17bm0804003h0001, 18bm0804003h0002, 19bm0804003h0003, 20bm0804003h0104, 20bm0804023h0001) from Japan Agency for Medical Research and development, AMED, by Grant-in-Aid for Scientific Research on Innovative Areas “Singularity Biology (No.8007)” (Grant Number 19H05428) of MEXT, by Grant-in-Aid for Scientific Research on Innovative Areas “Brain Protein Aging and Dementia Control” (Grant Number 17H05704) of MEXT, by Grant-in-Aid for Scientific Research (C) (Grant Number 20K06914) of JSPS, by The Naito Foundation, by GSK Japan Research Grant 2015, by the Science Research Promotion Fund of The Promotion and Mutual Aid Corporation for Private Schools of Japan , by Kitasato University School of Allied Health Sciences (Grant-in-Aid for Research Project) and by Graduate School of Medical Sciences, Kitasato University (Grant-in-Aid for Research Project, 2016-2017).
Publisher Copyright:
© 2020
PY - 2020/12
Y1 - 2020/12
N2 - 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.
AB - 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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U2 - 10.1016/j.scr.2020.102073
DO - 10.1016/j.scr.2020.102073
M3 - Article
C2 - 33181472
AN - SCOPUS:85095734865
SN - 1873-5061
VL - 49
JO - Stem Cell Research
JF - Stem Cell Research
M1 - 102073
ER -