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Research

Research
We’ve known for almost two decades that mutations in the MECP2 gene and its corresponding ...
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It’s been almost two decades since the discovery that alterations in MECP2 cause Rett ...
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Mutations in a single gene, MECP2, can be identified in the vast majority of individuals with ...
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The recent announcement that the gene therapy biotech company, AveXis, is advancing the program ...
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We are excited to report to the Rett Syndrome community and to everyone who cares about an ...
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Gene therapy is the lead program of our three-year strategic plan, Roadmap to a Cure. This ...
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RSRT has announced Roadmap to a Cure, a 3-year research plan featuring gene therapy as the lead ...
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Background In the early 1980s, few drugs were being developed for rare diseases as the small ...
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Rett syndrome-causing missense mutations cluster in two regions of MECP2. One of these regions ...
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We’ve tried to anticipate some of the questions that families will have about Roadmap to a ...
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RSRT was launched in 2008, in the wake of Adrian Bird’s remarkable reversal experiment. Our ...
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We live in exciting times. It seems like almost every day, breakthrough scientific discoveries ...
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Did you know an extra copy of MECP2 sleeps within each of our cells? Females have two X ...
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You -- the parents, grandparents, and families of children with Rett Syndrome -- know more than ...
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New hope for a devastating disease? Dangerous precedent for the FDA? Reactions are polarized to ...
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One of the hottest emerging approaches to altering or correcting defective disease-causing ...
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My inbox flooded with emails this past Wednesday following the announcement of the Chan ...
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Last week RSRT posted on Facebook an article about a gene therapy treatment for “bubble boy ...
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In previous work we did together with Dr. Jianrong Tang at our institute, we learned that deep ...
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Last year saw the exciting report that deep brain stimulation, a technique in which electrical ...
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Results from an RSRT funded clinical trial testing the drug copaxone (glatiramer acetate) in 10 ...
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While it’s true that Rett Syndrome is mainly a girls’ disorder, it’s not true that boys ...
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On July 11th we solicited questions on our blog for our Chief Scientific Officer, Randy ...
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Anyone who has ever driven a car knows the importance of counterbalancing the gas and brake ...