Dr. Huda Zoghbi has made critical contributions to the MECP2 Duplication Syndrome (MDS) field. Among the most important has been the discovery that reducing levels of the MeCP2 protein using an antisense oligonucleotide (ASO) strategy reverses symptoms in mouse models of MDS. She is working with the biotech company Ionis to develop an ASO therapeutic for MDS.

Dr. Zoghbi provides an update on the ASO work ongoing in her lab.
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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.

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