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

• 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
• Project Status: Completed (Fiscal Year 2014)
• Budget Amount: ¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000); Fiscal Year 2014: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000); Fiscal Year 2013: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000); Fiscal Year 2012: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
• Keywords: ライソゾーム病 / 再生医療 / 成育医療 / 糖鎖 / 先天性代謝異常症 / 細胞治療 / 間葉系幹細胞 / ライゾゾーム病 / 疾患モデルマウス

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