RNA Editing and Its Potential to Improve Rett Syndrome Symptoms | Rett Syndrome Research Trust

Описание: RNA editing, one of six genetic-based strategies that RSRT is pursuing for Rett syndrome, has seen explosive growth in the last handful of years and has generated attention from many academic labs and companies – and with good reason. A paper published in August 2022 online in Proceedings of the National Academy of Sciences shows that RNA editing can improve symptoms in a mouse model of Rett syndrome.

On August 25, 2022, the authors of that paper, Gail Mandel, PhD, and John Sinnamon, PhD, joined RSRT CEO Monica Coenraads for a discussion about what this discovery means for our progress toward a Rett syndrome cure.

Having received funding from RSRT for more than a decade, the Mandel lab at Oregon Health and Science University has put RNA editing on the map for Rett.
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The Rett Syndrome Research Trust is singularly focused on a cure for Rett syndrome. Achieving success takes a deep understanding of the relevant science, an environment built for scientific collaboration, and experience with drug and business development. Today we are building on incredible scientific and industry momentum by implementing six genetic-based strategies that tackle Rett from every angle. Every single company pursuing a cure for Rett is able to do so because they leveraged discoveries and resources that were generated with RSRT funding. RSRT is the home of the Rett cure community, and we are confident that together we will cure Rett syndrome.