Pharmacodynamic and Pharmacokinetic Properties of Full Phosphorothioate Small Interfering RNAs for Gene Silencing In Vivo

State-of-the-art small interfering RNA (siRNA) therapeutics such as givosiran and fitusiran are constructed from three variable components: a fully-modified RNA core that conveys metabolic stability, a targeting moiety that mediates target-cell uptake, and a linker. This structural complexity poses...

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Detalles Bibliográficos
Autores principales: Berk, Christian, Civenni, Gianluca, Wang, Yuluan, Steuer, Christian, Catapano, Carlo V., Hall, Jonathan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2021
Materias:
Original Papers
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8215415/
https://www.ncbi.nlm.nih.gov/pubmed/32311310
http://dx.doi.org/10.1089/nat.2020.0852

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8215415/
https://www.ncbi.nlm.nih.gov/pubmed/32311310
http://dx.doi.org/10.1089/nat.2020.0852

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