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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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Detalles Bibliográficos
Autores principales: Biscans, Annabelle, Caiazzi, Jillian, Davis, Sarah, McHugh, Nicholas, Sousa, Jacquelyn, Khvorova, Anastasia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
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