Ben Philpot, PhD, Bryan Roth, PhD, UNC | Antonio Bedalov, MD, PhD, Fred Hutchinson Cancer Research Center | Jeannie Lee, MD, PhD, Harvard University | Michael Green, MD, PhD, University of Massachusetts
Girls with Rett have a mutated copy of the MECP2 gene in every cell of their body. Fortunately, however, in every cell that is making mutated MeCP2 protein there is also a healthy copy of the MECP2 gene that is lying dormant. If we could wake up this silent copy we could, in theory, treat and maybe even cure Rett. RSRT is funding five labs in four universities who are working collaboratively to identify drugs that can reactivate the silent MECP2 gene.