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Bridging the Divide

October 11, 2017Research

We’ve known for almost two decades that mutations in the MECP2 gene and its corresponding protein, MeCP2, cause Rett Syndrome. During this time a variety of biological functions have been attributed to the MeCP2 protein, but with no clear evidence for how these various functions relate to Rett Syndrome. Years…

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Roadmap Update: Jaenisch Lab Award

September 20, 2017Research, Roadmap

In March of this year we announced our strategic research plan, Roadmap to a Cure, the cornerstone of which is four therapeutic approaches that we are undertaking in pursuit of a cure for Rett: gene therapy, MECP2 Reactivation, RNA editing/splicing, and protein replacement. Today’s announcement focuses on MECP2 Reactivation. A…

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What About Us?

July 12, 2017Research

It’s been almost two decades since the discovery that alterations in MECP2 cause Rett Syndrome. However, the diagnosis of Rett remains a clinical one based on a child’s symptoms and history. While a positive MECP2 genetic test confirms the diagnosis, it is not required. This creates a situation where individuals can…

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Precision Medicine

June 19, 2017Research

Mutations in a single gene, MECP2, can be identified in the vast majority of individuals with Rett Syndrome, yet each person is unique. As in any disease, treatments such as seizure or anxiety medications can be very successful for some individuals but not for others, even for those with the…

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AveXis Announcement – Delivering on Roadmap to a Cure

June 8, 2017Research, Roadmap

The recent announcement that the gene therapy biotech company, AveXis, is advancing the program generated by RSRT’s Gene Therapy Consortium is the most exciting news since Adrian Bird’s reversal paper a decade ago. First and foremost the future gene therapy clinical trial will be the first ever that addresses the…

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Gene Therapy Consortium Reports on Progress

May 15, 2017Research

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…

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Gene Therapy Primer

May 10, 2017Research

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…

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