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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...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Chemical Society
2023
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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 |