Gene editing of patient-derived iPS cells by Zinc finger nuclease to analyze development of juvenile myelomonocytic leukemia

Research Project

Project/Area Number	15K19177
Research Category	Grant-in-Aid for Young Scientists (B)
Allocation Type	Multi-year Fund
Research Field	Laboratory medicine
Research Institution	Shinshu University
Principal Investigator	MATSUDA Kazuyuki 信州大学, 医学部附属病院, 主任臨床検査技師 (00647084)
Project Period (FY)	2015-04-01 – 2017-03-31
Project Status	Completed (Fiscal Year 2016)
Budget Amount *help	¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000) Fiscal Year 2016: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000) Fiscal Year 2015: ¥2,990,000 (Direct Cost: ¥2,300,000、Indirect Cost: ¥690,000)
Keywords	ZFN / JMML / PTPN11 / 若年性骨髄単球性白血病 / iPS細胞 / ジンクフィンガーヌクレアーゼ / ヌクレオフェクション / 遺伝子改変
Outline of Final Research Achievements	We performed gene correction using Zinc Finger Nuclease (ZFN) specific for PTPN11 gene in juvenile myelomonocytic leukemia (JMML)-derived iPS cells with mutated PTPN11. PCR and direct sequencing confirmed that the mutated nucleotide was converted into wild-type one precisely. We will introduce mutation into PTPN11 wild-type iPS cells by the same protocol. The two types of gene-edited iPS cells (mutant-to-wild and wild-to-mutant) are useful for analysis of the cell kinetics to reveal the mechanism of development of JMML.

Report

(3 results)