Conjugation of small interfering RNA (siRNA) to an asialoglycoprotein receptor ligand
derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery of the siRNA to …

Covalent attachment of a synthetic triantennary N-acetylagalactosamine (GalNAc) ligand to
chemically modified siRNA has enabled asialoglycoprotein (ASGPR)-mediated targeted …

Asialoglycoprotein receptor (ASGPR) mediated delivery of triantennary N-acetylgalactosamine
(GalNAc) conjugated short interfering RNAs (siRNAs) to hepatocytes is a promising …

We recently demonstrated that siRNAs conjugated to triantennary N‐acetylgalactosamine (GalNAc)
induce robust RNAi‐mediated gene silencing in the liver, owing to uptake mediated …

Small interfering RNA (siRNA)‐mediated silencing requires siRNA loading into the RNA‐induced
silencing complex (RISC). Presence of 5′‐phosphate (5′‐P) is reported to be critical …

ATTR amyloidosis is a systemic, debilitating and fatal disease caused by transthyretin (TTR)
amyloid accumulation. RNA interference (RNAi) is a clinically validated technology that may …

The hepatocyte-specific asialoglycoprotein receptor (ASGPR) is an ideal candidate for targeted
drug delivery to the liver due to its high capacity for substrate clearance from circulation …

Non-alcoholic fatty liver disease (NAFLD) is a liver manifestation of metabolic syndrome,
and is estimated to affect one billion individuals worldwide. An increased intake of a high-fat …

A critical challenge for the successful development of RNA interference-based therapeutics
therapeutics has been the enhancement of their in vivo metabolic stability. In therapeutically …

Although 2′-deoxy-2′-α-F-2′-β-C-methyl (2′-F/Me) uridine nucleoside derivatives are a
successful class of antiviral drugs, this modification had not been studied in oligonucleotides…