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

2012 Fiscal Year Final Research Report

• 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
• Keywords: 再生医療 / ライソゾーム病

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.