Establishment of the iPS cells derived from the paroxysmal nocturnal hemoglobinuria clone cells and its application to a study of bone marrow failure.

Research Project

Project/Area Number	25860785
Research Category	Grant-in-Aid for Young Scientists (B)
Allocation Type	Multi-year Fund
Research Field	Hematology
Research Institution	The University of Tokyo
Principal Investigator	WATADANI Kumi 東京大学, 医学部附属病院, 助教 (70550432)
Project Period (FY)	2013-04-01 – 2015-03-31
Project Status	Completed (Fiscal Year 2014)
Budget Amount *help	¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000) Fiscal Year 2014: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000) Fiscal Year 2013: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Keywords	発作性夜間色素性尿症 / iPS細胞 / 発作性夜間ヘモグロビン尿症 / 多能性幹細胞 / PNH
Outline of Final Research Achievements	Establishment of induced pluripotent stem (iPS) cells derived from the affected cells of bone marrow failure including paroxysmal nocturnal hemoglobinuria is under investigation now. The object of this study is to establish the disease-specific iPS cells derived from PNH clone cells effectively and to promote elucidating the etiology of the disease by analyzing the hematopoietic cells differentiated from the iPS cells. We conducted trying induce iPS cells by reprogramming the PNH clone cells with CD55 negative and CD 59 negative populations of the bone marrow or the peripheral blood of classical PNH cases by use of Sendai virus vectors and non-viral episomal vectors.