Supported projects

N202004 – Therapeutic approaches by modulating mRNA splicing for the treatment of neuromuscular diseases

Scientific leader
Dr. Aurélie Goyenvalle

Our research projects aim to develop innovative therapeutic approaches for the treatment of neuromuscular diseases, such as Duchenne muscular dystrophy or spinal muscular atrophy. These strategies are based on modulation of gene expression using small nucleic acid sequences called “antisense oligonucleotides”. Our laboratory has been developing for several years a new generation of antisense oligonucleotides which have demonstrated unique properties in terms of biodistribution. As part of the preclinical development of these new molecules, it is crucial to be able to measure the therapeutic benefit through the precise quantification of the restored proteins. This decisive quantification can only be carried out using advanced equipment, such as the “ProteinSimple capillary immunoassay Jess” system.



Team 1 Biothérapies des maladies neuromusculaires

Université de Versailles Saint-Quentin-en-Yvelines

2 Avenue de la source de la bièvre

78 180 Montigny-le-Bretonneux