ClinASO: An open-source platform for rapid drug discovery of gapmer antisense oligonucleotides

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Shunkai Chen, Hao Liu, Dezi Cong, Ao Dong, Yuhang Wang, Jingyi Bi, Shijie Guo, Juan Yang, Xiaolei Wang, Guiping Ren, Ke Zhang, Haisheng Wang
+2 more Fan Lai, Yunkun Dang
DOI: 10.1016/j.omtn.2026.102933
Cited by: 1
References: 70

Abstract

genes implicated in metabolic dysfunction-associated liver disease (MASLD). In multiple human cells, the ASO achieved robust silencing effect. Notably, by conjugating GalNAc, this ASO demonstrated durable, liver-specific knockdown, significantly ameliorating hepatic steatosis and normalizing systemic lipid profiles in MASLD mouse model. Together, these findings establish ClinASO as an efficient, experimentally validated online tool for the rational design and rapid development of translatable ASO therapeutics.

Fields of Study & Subjects

DNA and Nucleic Acid Chemistry Advanced biosensing and bioanalysis techniques Genomics and Chromatin Dynamics

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Publisher
Elsevier BV
Volume
37
Issue
2
Pages
102933–102933
Publication date
17 April 2026
Language
EN