Improvement of murine mucopolysaccharidosis type VII by intramuscular transplantation of marrow-derived cells

Research Project

Project/Area Number	21791023
Research Category	Grant-in-Aid for Young Scientists (B)
Allocation Type	Single-year Grants
Research Field	Pediatrics
Research Institution	National Research Institute for Child Health and Development
Principal Investigator	TANAKA Toju 独立行政法人国立成育医療研究センター, 生殖・細胞医療研究部, 研究員 (50415585)
Project Period (FY)	2009 – 2012
Project Status	Completed (Fiscal Year 2012)
Budget Amount *help	¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000) Fiscal Year 2012: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000) Fiscal Year 2011: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000) Fiscal Year 2010: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000) Fiscal Year 2009: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000)
Keywords	再生医療 / ライソゾーム病 / 酵素活性 / 疾患モデルマウス / 細胞治療 / ムコ多糖症 / 先天性代謝異常症 / 再生医学 / トランスレーショナルリサーチ / 発生・分化 / 医療・福祉
Research Abstract	The therapeutic efficacy of cell transplantation for muscular lesions in lysosomal storage disorders was explored using a murine model of mucopolysaccharidosis type VII (MPS VII). We used stromal cells derived from bone marrow and expanded in vitro as the source of graft materials. We transplanted marrow stromal cells into the muscular tissue of MPS VII mice and found that donor cells migrated far beyond the site of injection. The GUSB activity was recovered after transplantation. In addition, histological analysis revealed a widespread decrease in lysosomal storage in the recipients. These results suggest that intramuscular transplantation of marrow stromal cells is feasible for treatment of lesions associated with lysosomal storage disorders.