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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...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Biscans, Annabelle, Caiazzi, Jillian, Davis, Sarah, McHugh, Nicholas, Sousa, Jacquelyn, Khvorova, Anastasia
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7430635/
https://ncbi.nlm.nih.gov/pubmed/32672813
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkaa595
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