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Precise Targeted Cleavage of a r(CUG) Repeat Expansion in Cells by Using a Small-Molecule–Deglycobleomycin Conjugate
RNA repeat expansions cause more than 30 neurological and neuromuscular diseases with no known cures. Since repeat expansions operate via diverse pathomechanisms, one potential therapeutic strategy is to rid them from disease-affected cells, using bifunctional small molecules that cleave the aberran...
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| Publicado no: | ACS Chem Biol |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7360342/ https://ncbi.nlm.nih.gov/pubmed/32186845 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acschembio.0c00036 |
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