Gene Therapy Consortium Reports on Progress
May 15, 2017
We are excited to report to the Rett Syndrome community and to everyone who cares about an afflicted family on recent progress made by RSRT’s Gene Therapy Consortium. As you may know, Rett Syndrome is caused by spontaneous mutations in the MECP2 gene that prevents the production of healthy MeCP2…
Gene Therapy Primer
May 10, 2017
Gene therapy is the lead program of our three-year strategic plan, Roadmap to a Cure. This potential one-time treatment, which targets Rett at its very root, has produced dramatic results in mouse models of the disease, more so, by far, than any other drug treatment studied thus far. Let’s review…
Racing Down The Gene Therapy Highway To A Cure
April 25, 2017
RSRT has announced Roadmap to a Cure, a 3-year research plan featuring gene therapy as the lead program. Gene therapy fixes the cause of a genetic disorder instead of alleviating the symptoms, essentially representing a cure. The theory is simple – replace defective genes with healthy copies. However, it is…
Orphan Drug Designation: What Does it Mean?
March 30, 2017
Background In the early 1980s, few drugs were being developed for rare diseases as the small number of patients made it difficult for companies to recoup the costs of drug development, let alone make a profit. With an estimated 7,000 rare diseases affecting 25-30 million Americans, Congress determined that laws…
New Insight Into MECP2 Mutation Hotspot
March 28, 2017
Rett syndrome-causing missense mutations cluster in two regions of MECP2. One of these regions – the methyl-CpG binding domain (MBD) – has been extensively characterized and helps MeCP2 bind DNA. The other region is called the NCoR/SMRT interaction domain (NID) and recruits a gene-silencing complex, but its function has remained comparatively…
Roadmap to a Cure FAQ
March 24, 2017
We’ve tried to anticipate some of the questions that families will have about Roadmap to a Cure. What if you don’t raise the full $33 million? Failure is not an option. We simply must raise the full amount. In fact, there are a number of other worthy efforts that merit…
RSRT Awards $7.5 Million to Research in 2016
March 17, 2017
RSRT was launched in 2008, in the wake of Adrian Bird’s remarkable reversal experiment. Our mission has always been a cure, not an improvement of some degree in a symptom or two… an all-out cure. Our 2016 awards, totaling $7.5 million, reflect our mission. RESEARCH AWARDED BY YEAR: Over $2…
