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   Articles with "galnac sirna" as a keyword



Differential Protein Precipitation-Based GalNAc-siRNA Sample Preparation with LC/MS Method Development Workflow in Plasma

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recommendations! Published in 2025 at "Analytical Chemistry"

DOI: 10.1021/acs.analchem.5c01587

Abstract: Liquid chromatography–mass spectrometry (LC/MS) plays a crucial role in the quantification of small interfering RNAs (siRNAs) in biological matrices. However, the recovery of siRNA from complex biological matrices remains a significant challenge. Focusing on liver-targeted… read more here.

Keywords: method; protein precipitation; differential protein; galnac sirna ... See more keywords

Metabolically Stable Anomeric Linkages Containing GalNAc-siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways.

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recommendations! Published in 2023 at "Journal of medicinal chemistry"

DOI: 10.1021/acs.jmedchem.2c01337

Abstract: Conjugation of synthetic triantennary N-acetyl-d-galactosamine (GalNAc) to small interfering RNA (siRNA) mediates binding to the asialoglycoprotein receptor (ASGPR) on the surface of hepatocytes, facilitating liver-specific uptake and siRNA-mediated gene silencing. The natural β-glycosidic bond of… read more here.

Keywords: anomeric linkages; sirna; galnac sirna; glycosidase ... See more keywords

GalNac-siRNA conjugate delivery technology promotes the treatment of typical chronic liver diseases

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recommendations! Published in 2025 at "Expert Opinion on Drug Delivery"

DOI: 10.1080/17425247.2025.2466767

Abstract: ABSTRACT Introduction Nucleic acid-based therapeutics have become a key pillar of the ‘third wave’ of modern medicine, following the eras of small molecule inhibitors and antibody drugs. Their rapid progress is heavily dependent on delivery… read more here.

Keywords: technology; galnac sirna; delivery; liver diseases ... See more keywords

From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization

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recommendations! Published in 2022 at "Nucleic Acids Research"

DOI: 10.1093/nar/gkac539

Abstract: Abstract Preclinical mechanistic studies have pointed towards RNA interference-mediated off-target effects as a major driver of hepatotoxicity for GalNAc–siRNA conjugates. Here, we demonstrate that a single glycol nucleic acid or 2′–5′-RNA modification can substantially reduce… read more here.

Keywords: safety; seed pairing; sirna conjugates; galnac sirna ... See more keywords

The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates

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recommendations! Published in 2022 at "Frontiers in Pharmacology"

DOI: 10.3389/fphar.2022.1090237

Abstract: RNA interference has become increasingly used for genetic therapy following the rapid development of oligonucleotide drugs. Significant progress has been made in its delivery system and implementation in the treatment of target organs. After a… read more here.

Keywords: prospects acetylgalactosamine; sirna conjugates; sirna; therapeutic prospects ... See more keywords