Ribofuranose-based GalNAc-conjugated siRNA enhances the liver-targeted delivery and elicits robust RNAi-mediated gene silencing

The N-acetylgalactosamine (GalNAc)-conjugate delivery platform has emerged as a pivotal enabling technology for the clinical translation of oligonucleotide therapeutics. The development of GalNAc-conjugated small interfering RNA (siRNA) therapeutics has been significantly advanced through structural...

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Bibliografische Detailangaben

Hauptverfasser:	Ze-Ao Huang, Zhen-Min Li, Jie Wang, Guang-Shen Ji, Zhong-Cai Gao, Ai-Fu Chen, Zhi-Kang Tian, Fei Yu, Geng-Shen Song
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	Elsevier 2026-03-01
Schriftenreihe:	Molecular Therapy: Nucleic Acids
Schlagworte:	MT: Delivery Strategies N-acetylgalactosamine GalNAc-siRNA conjugates liver-targeted delivery ribofuranose ring deoxyribofuranose ring
Online-Zugang:	http://www.sciencedirect.com/science/article/pii/S2162253125003555
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