Assessing biological activity and in vivo efficiency of the epigenetic activator, 3R-ON

Investigators: Gregory J Block MSc PhD, Shiri Levy PhD

Category: Research - Basic

We have previously proposed treating FSHD by upregulating genes that turn DUX4 off. This approach is unique from other strategies that silence DUX4 directly; however, we believe this approach will create a more feasible therapeutic modality for FSHD patients. We are exploring two ways of upregulating FSHD-related genes. The first is using our EB-ON technology, which “removes the brakes” from a gene. The second is our 3R-ON technology, which “applies the gas”. In this grant we seek to exploit the surprising finding that 3R-ON is uniquely potent, and thus could be used to measure different ways of delivering the mRNA into the muscle fiber. Understanding delivery is a key to success for developing an mRNA medicine for FSHD.