Introducing an In Vitro Liver Stability Assay Capable of Predicting the In Vivo Pharmacodynamic Efficacy of siRNAs for IVIVC

There has been a renewed interest in therapeutic small interfering RNAs (siRNAs) over the past few years. This is particularly the result of successful and efficient delivery of N-acetylgalactosamine (GalNAc)-conjugated siRNAs to the liver. In general, the lead selection process for siRNA drugs is f...

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Bibliografiske detaljer
Udgivet i:Mol Ther Nucleic Acids
Main Authors: Basiri, Babak, Xie, Fang, Wu, Bin, Humphreys, Sara C., Lade, Julie M., Thayer, Mai B., Yamaguchi, Pam, Florio, Monica, Rock, Brooke M.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7415771/
https://ncbi.nlm.nih.gov/pubmed/32771924
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.07.012
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