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Recent discoveries provide us with a clearer understanding of how the genetics of FSHD translate into disease. This new information defines a target for therapy development. We will both engineer a model and use existing models to test a novel therapeutic approach called RNA interference to eliminate disease that attacks messenger RNA in the cell to prevent disease causing protein production. The FSHD protein is called DUX4 and leads to muscle damage. Our therapeutic approach will reduce production of toxic protein to eliminate the FSHD muscle damage with a single application to muscles throughout the body. We have been working on this approach in other animal models of muscular dystrophy, and what we have learned from these studies will be applied to new FSHD mouse models based on DUX4 protein expression for development of a potential treatment for FSHD.
The cell pathway (RNA interference or RNAi) that I am using to target and destroy DUX4 messenger RNA (mRNA) to prevent toxic DUX4 protein as a therapeutic approach for FSHD.
— Joel Chamberlain