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

2016 Fiscal Year Final Research Report

• 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
• Keywords: ZFN / JMML / PTPN11

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.

Free Research Field

遺伝子・染色体解析