* Interest of the canine models in hereditary myopathies 

* Characterization of hereditary  myopathies

* Research of therapeutic processes

* Development of tools of functional evaluations

unit of study and therapy of myopathies

The myopathies are affections characterized by a degeneration of the muscular tissue, the most known being the Duchenne myopathy or Duchenne Muscular Dystrophy (DMD). This is a genetic disease linked to the X chromosome, which affects one boy out of 3500. It results from mutations in the gene encoding the dystrophin, a monumental protein (427 kD). Normally localised under the muscle fibre membrane, the dystrophin connects the cytoskeleton to the basal lamina. In patients affected by dystrophinopathy (pool of diseases linked to dystrophin anomalies), the absence or the dysfunction of the dystrophin weakens the muscle fibre membrane. It results from that, cycles of necroses and regenerations which gradually exhaust the muscular regeneration capacities. Currently, there is no curative treatment of the disease. However, novel therapeutic strategies, involving cellular, genic or pharmacological tools, are promising.

The Unit of Study and Therapy of Myopathies is directed by Stéphane Blot, and studies canine and feline hereditary myopathies. Then, using phenotypic and molecular characterizations of these diseases, the UETM adapts to the affected dogs therapeutic strategies having already proven their abilities in vitro or in small-size animal models. Thereby, the UETM undertakes preclinical researches. In order to evaluate the efficiency of the performed strategies, the group develops tools of functional evaluations, often applicable to the human patient.