A new approach to gene therapy for Steinert’s disease
Myology Institute's team has developed and tested a new approach to gene therapy, a so-called “decoy” approach, for Steinert’s disease or myotonic dystrophy type 1.
The Denis Furling team (Paris, France), in collaboration with the Nicolas Sergeant team (Lille, France), has developed and tested a new approach to gene therapy, a so-called “decoy” approach, for Steinert’s disease or myotonic dystrophy type 1 (DM1).
The strategy is based on the expression of modified proteins that will bind to the pathological expansions of the mutated RNAs in the location of and instead of the endogenous MBNL1 proteins, in order to free the latter and restore their activity in the muscle cells expressing the DM1 mutation.
The results have just been published in the Nature Biomedical Engineering Journal.