Whole-Body Physiologically-Based Pharmacokinetic Modeling of GalNAc conjugated siRNAs

Abstract excerpt

<title>Abstract</title> <p><bold>Introduction</bold> N-Acetyl-galactosamine small interfering RNAs (GalNAc-siRNA) is an emerging class of drugs due to their durable knockdown of disease related proteins. Conjugation onto GalNAc allows for target specific uptake into hepatocytes via the Asia Glycoprotein Receptor (ASGPR). With a transient plasma exposure combined with a rapid liver uptake and prolonged half-life i...

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• Amyloid Neuropathies, Familial
• Amyloidosis: Diagnosis, Treatment, Outcomes
• CRISPR and Genetic Engineering
• Drug Carriers
• Drug Dosage Calculations
• Drug Transport and Resistance Mechanisms
• MicroRNA in disease regulation
• RNA Interference and Gene Delivery

Identifiers and source

Literature Corpus work

49b4a2f8-fbac-5aca-8325-1dec8ee2f54f

DOI

10.21203/rs.3.rs-4691195/v1