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Structure of the Myotonic Dystrophy Type 2 RNA and Designed Small Molecules That Reduce Toxicity
Myotonic dystrophy type 2 (DM2) is an untreatable neuromuscular disorder caused by a r(CCUG) expansion (r(CCUG)(exp)) that folds into an extended hairpin with periodically repeating 2×2 nucleotide internal loops (5’CCUG/3’GUCC). We designed multivalent compounds that improve DM2-associated defects u...
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| Hoofdauteurs: | , , , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
2013
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3944380/ https://ncbi.nlm.nih.gov/pubmed/24341895 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/cb4007387 |
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