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Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)(exp) Degrader in Myotonic Dystrophy

[Image: see text] Myotonic dystrophy type 1 (DM1) is caused by a highly structured RNA repeat expansion, r(CUG)(exp), harbored in the 3′ untranslated region (3′ UTR) of dystrophia myotonica protein kinase (DMPK) mRNA and drives disease through a gain-of-function mechanism. A panel of low-molecular-w...

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Detalles Bibliográficos
Autores principales:	Gibaut, Quentin M. R., Bush, Jessica A., Tong, Yuquan, Baisden, Jared T., Taghavi, Amirhossein, Olafson, Hailey, Yao, Xiyuan, Childs-Disney, Jessica L., Wang, Eric T., Disney, Matthew D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Chemical Society 2023
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10375898/ https://www.ncbi.nlm.nih.gov/pubmed/37521782 http://dx.doi.org/10.1021/acscentsci.2c01223

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