Mechanistic Pharmacokinetics and Pharmacodynamics of GalNAc-siRNA: Translational Model Involving Competitive Receptor-Mediated Disposition and RISC-Dependent Gene Silencing Applied to Givosiran
Vivaswath S. Ayyar, Dawei Song
Topics & Concepts
Gene silencingSmall interfering RNAIntracellularPharmacokineticsPharmacologyRNAChemistryMetabolitePharmacodynamicsCell biologyBiophysicsBiologyBiochemistryGeneRNA Interference and Gene DeliveryRNA regulation and diseaseRNA and protein synthesis mechanisms