Development of A Cell Based Therapy for Animal Models for Lysosomal Storage Disorder

2014 Fiscal Year Final Research Report

• Project/Area Number: 24659513
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Embryonic/Neonatal medicine
• Research Institution: National Research Institute for Child Health and Development
• Principal Investigator: OHKURA TAKASHI 独立行政法人国立成育医療研究センター, 再生医療センター, 研究員 (50183223)
• Co-Investigator(Kenkyū-buntansha): UMEZAWA Akihiro 独立行政法人国立成育医療研究センター 再生医療センター, センター長 (70213486)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Keywords: ライソゾーム病

Outline of Final Research Achievements

Instead of enzyme replacement therapy (ERT) for lysosomal storage diseases (LDSs), cell replacemant therapy of mesenchymal stem cells or iPS cells is estimated its validity and safety using Sandhoff disease (SD) mouse. Urinaly oligosaccharides of SD mouse were tritium-labelled and analysed structurally using lectin affinity chromatography and HPLC. It contained the same oligosaccharides, GlcNAc1-2-Man3-GlcNAcOT as human’s one. It was indicated that serum enzyme activity of SD-scid mouse was recovered 5-15% of wild type by transplantation of iPS cells

Free Research Field

糖鎖生物学