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Targeting the r(CGG) Repeats That Cause FXTAS with Modularly Assembled Small Molecules and Oligonucleotides
[Image: see text] We designed small molecules that bind the structure of the RNA that causes fragile X-associated tremor ataxia syndrome (FXTAS), an incurable neuromuscular disease. FXTAS is caused by an expanded r(CGG) repeat (r(CGG)(exp)) that inactivates a protein regulator of alternative pre-mRN...
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| Veröffentlicht in: | ACS Chem Biol |
|---|---|
| Hauptverfasser: | , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
American
Chemical
Society
2014
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| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4287843/ https://ncbi.nlm.nih.gov/pubmed/24506227 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/cb400875u |
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