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Progress made in treating Duchenne muscular dystrophy

Researchers have been able to improve muscle strength in dogs suffering from Duchenne muscular dystrophy, an achievement that opens the door to possible treatments for people with this rare genetic disease.

Dog suffering from Duchenne muscular dystrophy. © INRA
Updated on 12/02/2014
Published on 11/13/2014

Researchers from the gene therapy laboratory in Nantes injected a “medicinal gene” in the paws of 18 dogs suffering from Duchenne muscular dystrophy. The team of pathologists and technicians from the UMR 703 Joint Research Unit then looked at how the animals’ muscle tissue reacted to evaluate the beneficial effects of the treatment. This research project also called on the expertise from the Boisbonne Gene and Cell Therapy Centre.

The Joint ONIRIS-INRA Research Unit for Animal Pathophysiology and Biotherapies for Muscle and Nervous System Disorders. © INRA
The Joint ONIRIS-INRA Research Unit for Animal Pathophysiology and Biotherapies for Muscle and Nervous System Disorders © INRA

Tissue. © INRA
Tissue © INRA
“After three and a half months, tests showed that the muscular degeneration had been blocked in the treated paw,” says Caroline Le Guiner, a researcher from Atlantic Gene Therapies who coordinated the study published in the medical journal Molecular Therapy. In dogs receiving the highest dose, 80% of the muscle fibres were repaired to be able to produce dystrophin protein, “whereas just 40% is enough to improve muscle strength,” added Le Guiner.

The treatment used the technique of “exon skipping”, which consists in ignoring faulty sections of genetic code, to send a shortened but coherent message that partially re-established production of the missing protein. Exons are gene encoding fragments.

The next step will be to begin a clinical trial on a small number of people with this disease. Patients will initially receive a local injection in the arm in an attempt to regain muscle strength.

Partnership
This gene therapy is the result of several years of research by the Atlantic Gene Therapies Institute, composed of the INSERM UMR 1089 Joint Research Unit and INRA/ONIRIS UMR 703 Joint Research Unit (Angers/Nantes centre). The Institute receives support from the Pays de la Loire region, Centre de Référence des Maladies Neuromusculaires Rares, Centres Hospitalo-Universitaires of Nantes and Angers and the Association Française contre les Myopathies (AFM).

Reference
Le Guiner, C., Montus, M., Servais, L., Cherel, Y., Francois, V., Thibaud, J. L., Wary, C., Matot, B., Larcher, T., Guigand, L., Dutilleul, M., Domenger, C., Allais, M., Beuvin, M., Moraux, A., Le Duff, J., Devaux, M., Jaulin, N., Guilbaud, M., Latournerie, V., Veron, P., Boutin, S., Leborgne, C., Desgue, D., Deschamps, J. Y., Moullec, S., Fromes, Y., Vulin, A., Smith, R. H., Laroudie, N., Barnay-Toutain, F., Riviere, C., Bucher, S., Le, T. H., Delaunay, N., Gasmi, M., Kotin, R. M., Bonne, G., Adjali, O., Masurier, C., Hogrel, J. Y., Carlier, P., Moullier, P., & Voit, T. (2014). Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients. Molecular Therapy: the journal of the American Society of Gene Therapy.  DOI: 10.1038/mt.2014.151

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Animal Health
Associated Centre(s):
Angers-Nantes

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Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a rare disease that affects 150 to 200 newborn baby boys in France each year, or one birth every three days. The disease is caused by a deficient gene that blocks the production of dystrophin, a protein essential for muscle function. In France, some 2500 people are affected by this disease, which is characterised by general, progressive and irreversible muscle weakness. Currently, there is no treatment available that can reverse the disease’s effects.

The Joint Research Unit (ONIRIS-INRA) for Animal Pathophysiology and Biotherapies for Muscle and Nervous System Disorders

This unit’s research is focused on the creation of therapeutic strategies for genetic diseases affecting the muscles (Duchenne muscular dystrophy) or the nervous system (type II glycogen storage disease, also known as Pompe disease). The unit’s gene and cell therapies are developed using large animal models and aim to identify relevant therapeutic approaches by demonstrating their efficacy and safety before clinical trials are begun.
The unit includes an expert team in anatomical pathology, called APEX, whose chief mission is to lend its expertise on animal histopathology and tissue and cell phenotyping to research teams. It also helps characterise animal models and improve the understanding of the underlying pathogenic mechanisms of genetic or infectious diseases. In addition, it monitors information released about emerging or re-emerging diseases.