Cell therapeutic approach using gene-transduced mesenchymal stromal cells for muscular dystrophy

2016 Fiscal Year Final Research Report

• Project/Area Number: 26860273
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Experimental pathology
• Research Institution: Nippon Medical School (2016); National Center of Neurology and Psychiatry (2014-2015)
• Principal Investigator: YUKO KASAHARA 日本医科大学, 大学院医学研究科, 助教 (90391911)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 間葉系幹細胞 / 筋ジストロフィー

Outline of Final Research Achievements

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease that causes respiratory and cardiac failure. Inflammation is involved in the pathogenesis of muscular dystrophy and therefore immunosuppressants can be utilized to improve muscle function. For cell therapeutic approach, multipotent mesenchymal stromal cells (MSCs) are promising because of their immunosuppressive properties and ability to differentiate into myogenic-lineage. In this study, we demonstrated that the correlations of inflammation with the severity of DMD by the results of dystrophic mice, which exhibited more severe inflammation and cardiorespiratory dysfunction by genetic ablation of IL-10. We established safe and effective cell transplantation of MSCs into DMD dogs and also indicated the effects of IL-10 on the MSCs engraftment in the skeletal muscle. The strategy would be promising for the anti-inflammatory therapy of DMD, although further study is required for the mechanism of interaction.

Free Research Field

細胞生物学、生化学、分子生物学