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Combination Treatment of Erythromycin and Furamidine Provides Additive and Synergistic Rescue of Mis-splicing in Myotonic Dystrophy Type 1 Models
[Image: see text] Myotonic dystrophy type 1 (DM1) is a multisystemic disease that presents with clinical symptoms including myotonia, cardiac dysfunction, and cognitive impairment. DM1 is caused by a CTG expansion in the 3′ UTR of the DMPK gene. The transcribed expanded CUG-repeat RNA sequester the...
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| Publicado no: | ACS Pharmacol Transl Sci |
|---|---|
| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American
Chemical Society
2019
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| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6726129/ https://ncbi.nlm.nih.gov/pubmed/31485578 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acsptsci.9b00020 |
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