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Targeted splice sequencing reveals RNA toxicity and therapeutic response in myotonic dystrophy

Biomarker-driven trials hold promise for therapeutic development in chronic diseases, such as muscular dystrophy. Myotonic dystrophy type 1 (DM1) involves RNA toxicity, where transcripts containing expanded CUG-repeats (CUG(exp)) accumulate in nuclear foci and sequester splicing factors in the Muscl...

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Detalles Bibliográficos
Autores principales:	Tanner, Matthew K, Tang, Zhenzhi, Thornton, Charles A
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	RNA and RNA-protein complexes
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7913682/ https://www.ncbi.nlm.nih.gov/pubmed/33503262 http://dx.doi.org/10.1093/nar/gkab022

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