ClinASO: An open-source platform for rapid drug discovery of gapmer antisense oligonucleotides
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.
Authors
- Yunkun Dang (ORCID: https://orcid.org/0000-0002-8206-0271)
- Shunkai Chen
- Dezi Cong (ORCID: https://orcid.org/0000-0002-5692-235X)
- Shijie Guo
- Jingyi Bi
- Ke Zhang
- Xiaolei Wang
- Hao Liu
- Juan Yang
- Ao Dong
- Yuhang Wang
- Fan Lai
- Guiping Ren
- Haisheng Wang
Institutions
- Yunnan University (CN)
- Kunming Medical University (CN)
- Jiangxi Science and Technology Normal University (CN)
- Bioscience Research (US)
- Shenzhen Bay Laboratory (CN)
- First Affiliated Hospital of Kunming Medical University (CN)
- Shenzhen Academy of Aerospace Technology (CN)
- University of Chinese Academy of Sciences (CN)
- Lanzhou University (CN)
Publication Details
- Journal
- Molecular Therapy — Nucleic Acids
- Published
- 2026-04-17
- DOI
- https://doi.org/10.1016/j.omtn.2026.102933
- Primary Topic
- DNA and Nucleic Acid Chemistry
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Natural Science Foundation of China
- Yunnan Provincial Science and Technology Department
- National Key Research and Development Program of China Stem Cell and Translational Research