DYNACURE
A new strategy for the therapy of centronuclear myopathies
Project description
This project concerns the development of a therapeutic strategy for Central Nuclear Muscular Dystrophy (CNM), a group of very rare and serious myopathies for which no therapy currently exists.
Following the team's initial discovery of the role of Dynamine 2 in one of the 3 CNMs, a Conectus Alsace premature investment was made. All the work carried out by Jocelyn Laporte's team has shown that the decrease, by genetic crossbreeding, in the expression level of Dynamine 2, allows the survival of the mouse models of 2 of the CNMs, and has also allowed the development of an exogenous strategy to decrease the level of Dynamine 2 (by shRNA).
Why does Conectus invest in this project?
Conectus' financial investment will make it possible to carry out in vivo proof of concept of several exogenous strategies aimed at reducing the Dynamin 2 rate (shRNA, exon-skipping and antisense oligonucleotides) in the mouse models of 2 CNMs.
At the origin of the project:
Jocelyn Laporte, team leader of the "Physiopathology of Neuromuscular Diseases" team at IGBMC.
Focus
Conectus is investing in this project: €379,407
Duration: 18 months