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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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Detalles Bibliográficos
Autores principales: Basiri, Babak, Xie, Fang, Wu, Bin, Humphreys, Sara C., Lade, Julie M., Thayer, Mai B., Yamaguchi, Pam, Florio, Monica, Rock, Brooke M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7415771/
https://www.ncbi.nlm.nih.gov/pubmed/32771924
http://dx.doi.org/10.1016/j.omtn.2020.07.012