4.2 Article

Generation of gene-corrected iPSCs line (KETUi001-A) from a PARK8 patient iPSCs with familial Parkinson's disease carrying the I2020T mutation in LRRK2

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STEM CELL RESEARCH
卷 49, 期 -, 页码 -

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ELSEVIER
DOI: 10.1016/j.scr.2020.102073

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资金

  1. Regenerative Medicine (the Program for Intractable Disease Research Utilizing Disease-specific iPS Cells)
  2. Regenerative Medicine (Acceleration Program for Intractable Diseases Research Utilizing Disease-specific iPS Cells)
  3. Practical Research Project for Rare/Intractable Diseases from Japan Agency for Medical Research and development, AMED [16ek0109013h0003, 16ek0109158h0002, 16bm0609003h0005, 17bm0804003h0001, 18bm0804003h0002, 19bm0804003h0003, 20bm0804003h0104, 20bm0804023h0001]
  4. MEXT [8007, 19H05428, 17H05704]
  5. JSPS [20K06914]
  6. Naito Foundation
  7. GSK Japan Research Grant 2015
  8. Science Research Promotion Fund of The Promotion and Mutual Aid Corporation for Private Schools of Japan
  9. Kitasato University School of Allied Health Sciences
  10. Graduate School of Medical Sciences, Kitasato University
  11. Grants-in-Aid for Scientific Research [20K06914, 19H05428, 17H05704] Funding Source: KAKEN

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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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