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

doi:10.1016/j.scr.2020.102073

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

Research output: Contribution to journal › Article › peer-review

4 Citations (Scopus)

Abstract

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.

Original language	English
Article number	102073
Journal	Stem Cell Research
Volume	49
DOIs	https://doi.org/10.1016/j.scr.2020.102073
Publication status	Published - 12-2020
Externally published	Yes

All Science Journal Classification (ASJC) codes

Developmental Biology
Cell Biology

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

abstract = "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.",

author = "Etsuro Ohta and Takefumi Sone and Hideki Ukai and Tomoko Hisamatsu and Tokiko Kitagawa and Mitsuru Ishikawa and Makiko Nagai and Ueda, {Hiroki R.} and Fumiya Obata and Hideyuki Okano",

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year = "2020",

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doi = "10.1016/j.scr.2020.102073",

language = "English",

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journal = "Stem Cell Research",

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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

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

Abstract

All Science Journal Classification (ASJC) codes

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