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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...
Gorde:
| Argitaratua izan da: | Nucleic Acids Res |
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| Egile Nagusiak: | , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
Oxford University Press
2021
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7913682/ https://ncbi.nlm.nih.gov/pubmed/33503262 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkab022 |
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