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

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
Subjects:
In vitro in vivo correlation
IVIVC
siRNA
liver homogenate stability
mass spectrometry
metabolite identification
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120302018

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http://www.sciencedirect.com/science/article/pii/S2162253120302018

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