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Preparing for Gene Therapy Trials

November 17, 2017Research

A lot has happened in the six months since publication of our Gene Therapy Primer.  Notably, two gene therapies have been approved by the FDA (the first ever gene therapy approvals in the US) and a third has received a unanimous recommendation for approval from a panel of FDA advisors….

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Breakthrough Discoveries for DNA & RNA Editing

November 8, 2017Research

It’s been an exciting several weeks for the field of RNA and DNA editing. Three days after Gail Mandel’s team published their manuscript highlighted in a recent blog, “Editing out Rett Mutations,” researchers at MIT and Harvard also published manuscripts describing alternative methods similarly capable of correcting point mutations that…

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Editing Out Rett Mutations

October 26, 2017Research

Until we know what form the Rett cure will take it’s critical to pursue multiple strategies in parallel. So, in addition to providing the funding for the development of the first generation gene therapy for Rett Syndrome, now being pursued by AveXis, RSRT has also supported the parallel development of…

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RettGive: Easy, Impactful, Empowering

October 23, 2017Fundraising

I’m not the most tech-savvy person. My wife makes fun of me for fumbling with the TV remote, and my RSRT colleagues have probably good-naturedly rolled their eyes once or twice at my tech-related questions. So when we developed RSRT’s crowdfunding platform, RettGive, I was a little nervous about how…

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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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October: Rett Awareness and So Much More

October 10, 2017Community

I think most of us Rett parents compartmentalize our life events according to “pre-Rett” and “post-Rett”, I know I do. In my “post-Rett” life October carries significant importance. First and foremost my daughter Chelsea was born in October of 1996. October 10th to be exact. Today she turns 21 years…

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How Rett Changed a Granddad

October 4, 2017Community, Fundraising

Like other grandparents who faced a diagnosis of Rett Syndrome, I was initially devastated. My granddaughter Magnolia would not have the life I dreamed for her. I wouldn’t see her walk down an aisle or celebrate those typical life events most grandparents get to savor. The impact on my son,…

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