New cell therapy of Inborn error of metabolism

Research Project

Project/Area Number	15K09603
Research Category	Grant-in-Aid for Scientific Research (C)
Allocation Type	Multi-year Fund
Section	一般
Research Field	Pediatrics
Research Institution	Nihon University
Principal Investigator	ISHIGE Mika 日本大学, 医学部, 講師 (90420950)
Co-Investigator(Kenkyū-buntansha)	松本太郎日本大学, 医学部, 教授 (50366580) 谷ヶ崎博日本大学, 医学部, 准教授 (90378141)
Project Period (FY)	2015-04-01 – 2019-03-31
Project Status	Completed (Fiscal Year 2018)
Budget Amount *help	¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000) Fiscal Year 2017: ¥650,000 (Direct Cost: ¥500,000、Indirect Cost: ¥150,000) Fiscal Year 2016: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000) Fiscal Year 2015: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
Keywords	細胞治療 / 先天代謝異常症 / ライソソーム病 / 再生医療 / 間葉系幹細胞
Outline of Final Research Achievements	We examined the localization, phenotype and functional property of mesenchymal stem cells (MSC) and neural stem/progenitor cells in umbilical cord (UC). UC is very useful source of new cell therapy for the patient of inherited metabolic disease, particularly lysosomal disease, to neuronal regeneration, enhance engraftment of hematopoietic stem cells transplantation, and allogeneic cell-based therapy of immune disorders included graft-versus-Host disease (GVHD).
Academic Significance and Societal Importance of the Research Achievements	本研究により、臍帯動脈周囲組織由来の細胞は神経細胞の再生が期待できる移植ソースであること、臍帯組織には間葉系幹細胞（MSC）が存在し同部位由来MSCは免疫抑制機能が高く移植後の免疫反応抑制が期待できることが判明した。これより、臍帯は造血幹細胞移植を行う先天代謝異常症、特に神経再生を期待するライソソーム病の細胞治療に有用であると結論でき、新たな治療法の開発の可能性が高まった。