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The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy
Small interfering RNAs (siRNAs) have revolutionized the treatment of liver diseases. However, robust siRNA delivery to other tissues represents a major technological need. Conjugating lipids (e.g. docosanoic acid, DCA) to siRNA supports extrahepatic delivery, but tissue accumulation and gene silenci...
Autores principales: | Biscans, Annabelle, Caiazzi, Jillian, Davis, Sarah, McHugh, Nicholas, Sousa, Jacquelyn, Khvorova, Anastasia |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7430635/ https://www.ncbi.nlm.nih.gov/pubmed/32672813 http://dx.doi.org/10.1093/nar/gkaa595 |
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