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Therapeutic potential evaluation of umbilical cord and adipose tissue mesenchymal cells in calpain knockout mice

Grant number: 12/50198-9
Support Opportunities:Scholarships in Brazil - Post-Doctoral
Effective date (Start): June 01, 2012
Effective date (End): May 31, 2015
Field of knowledge:Biological Sciences - Morphology - Cytology and Cell Biology
Principal Investigator:Mayana Zatz
Grantee:Eder Zucconi
Host Institution: Instituto de Biociências (IB). Universidade de São Paulo (USP). São Paulo , SP, Brazil


Progressive Muscular Dystrophies (PMD) are clinically and genetically heterogeneous group of disorders for which there is no cure. The possibility to replace the defective muscle cells and improve muscular performance with cell therapy is a promising approach for the treatment of PMD, independently of the specific gene mutation. We recently compare the efficiency of two different sources of mesenchymal stromal cells (MSC) in muscle regeneration in vivo. When human adipose tissue (hASC) or human umbilical cord tissue (hUCT) MSC were injected in SJL mice (murine model for Limb Girdle Muscular Dystrophy 2B - LGMD2B), without immunosuppression, both MSC sources were able to reach the host muscle but surprisingly only hASC were able to express a significant amount of human muscle proteins and improve motor ability of injected animals. Therefore, preclinical studies in different animal models of muscular dystrophies are of utmost importance. Do hASC and hUCT MSC have a comparable behavior in different animal models of PMD? Here we propose to investigate the ability of hASC and hUCT-derived MSC for muscular regeneration in vivo in the calpain-3 knockout (C3KO) mice, murine model of LGMD2A. Calpain-3 (CAPN3) mutations accounts for the largest fraction of the autosomal recessive (AR) forms of LGMD and in the Brazilian population about 33% of AR LGMD families are caused by mutations in this gene. Although our experiments showed consistent results regarding the apparently better potential of hASC versus hUCT-derived MSC for muscle regeneration in vivo, we believe that preclinical studies in different animal models will be essential to confirm the present observations, which will have important implications before starting clinical trials in patients with PMD. (AU)

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