GRANT 31

This interdisciplinary research project aims to develop new polymeric carriers decorated with specific ligands for the transportation and the targeted delivery of antisense oligomers (AO) toward muscle cells as a therapeutic approach for neuromuscular disorders. AO’s appear as a promising strategy for hereditary neuromuscular disorders such as Duchenne Muscular Dystrophy, Myotonic Dystrophy and FSHD, but their use is still limited because of their rapid clearance from the bloodstream and their lack of tissue selectivity. Therefore this project will focus on the design of synthetic nanocarriers composed of functional polymers able to stabilize the AO against premature degradation and ensure its transportation and delivery to targeted muscle cells. The relevance of theses synthetic polymer vectors relies on their outstanding features such as i) biocompatibility, ii) biodegradability and iii) fine tuned functionality to overcome the biological barriers commonly met in gene therapy.