Progress Update: siRNA Based Compounds to Treat MECP2 Duplication Syndrome

Anastasia Khvorova, PhD, of University of Massachusetts Medical School presents an update on progress for a project she is undertaking as a potential treatment for MECP2 Duplication Syndrome. She is joined by her post-doctoral fellow, Vignesh Hariharan, PhD and host Monica Coenraads, CEO of the Rett Syndrome Research Trust.

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The Rett Syndrome Research Trust is singularly focused on a cure for Rett syndrome and related MECP2 disorders. 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.