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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Bibliographic Details
Main Authors: Babak Basiri, Fang Xie, Bin Wu, Sara C. Humphreys, Julie M. Lade, Mai B. Thayer, Pam Yamaguchi, Monica Florio, Brooke M. Rock
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Nucleic Acids
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Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120302018

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