Bone marrow transplantation as a therapeutic approach for dysferlinopathies
The aim of the study is to assess the therapeutic effect of bone marrow transplantation in A/J mice. So far, there is no effective treatment for dysferlinopathies. Preliminary studies from our group have shown some functional improvement in A/J mice treated with bone marrow transplantation. We have observed some functional improvements 22 weeks after bone marrow transplantation (BMT). We are interested in studying if this beneficial effect is maintained over time. To address this issue we have designed a new protocol in which animals will be analyzed at 22 weeks and 34 weeks of age (10 and 22 weeks after BMT) comparing the efficiency. We will study dysferlin expression in skeletal muscle and peripheral blood and perform functional studies i.e. electromyography and gait assessment.