GDF5: A potential treatment for (neuro)-muscular diseases

On April 19th at 2 p.m., an English-language seminar entitled “GDF5: a potential treatment for (neuro)-muscular diseases” will be held in Room 1 of the Monteggia Pavilion – Policlinico Hospital in Milan.

It will also be possible to participate online by clicking on the following link:

The speaker will be Prof. France Pietri-Rouxel, who has robust expertise in muscle biology, signal transduction and DMD pathophysiology. Team leader of “Gene Therapy for DMD & Skeletal Muscle Pathophysiology” in the Center of Myology, Faculté de Médecine de la Pitié Salpêtrière, Paris.


Among factors involved in muscle homeostasis, emerging relevance is observed for Growth Differentiation Factor 5 (GDF5) which has been shown to prevent muscle mass loss and force decline during ageing. We demonstrated that GDF5 overexpression induces muscle mass gain and improves neuromuscular connectivity and endplate morphology in a mouse model of Sarcopenia. GDF5 could also be a good candidate for preserving muscle mass in neuromuscular diseases as Duchenne muscular dystrophy (DMD). We investigated the role of GDF5 in DMD progression and showed that its over-expression modulates regeneration process and induces hyperplasia. Of relevance, we investigate the benefits of a combination of GDF5 with AAV-microdystrophin in improving gene therapy by preserving myofibers integrity and increasing muscle mass.


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