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Developing Bivalent Ligands to Target CUG Triplet Repeats, the Causative Agent of Myotonic Dystrophy Type 1
An expanded CUG repeat transcript (CUG(exp)) is the causative agent of myotonic dystrophy type 1 (DM1) by sequestering muscleblind-like 1 protein (MBNL1), a regulator of alternative splicing. Based on a ligand (1) that was previously reported to be active in an in vitro assay, we present the synthes...
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| Autori principali: | , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2013
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3925341/ https://ncbi.nlm.nih.gov/pubmed/24188018 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/jm400794z |
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