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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...
Autores principales: | Tran, Tuan, Childs-Disney, Jessica L., Liu, Biao, Guan, Lirui, Rzuczek, Suzanne, Disney, Matthew D. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American
Chemical
Society
2014
|
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4287843/ https://www.ncbi.nlm.nih.gov/pubmed/24506227 http://dx.doi.org/10.1021/cb400875u |
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