Work

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

Year: 2026

Type: article

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.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2026.102933

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, Fan Lai, Yunkun Dang

Institutions: Yunnan University, First Affiliated Hospital of Kunming Medical University, Lanzhou University, Shenzhen Bay Laboratory, Shenzhen Medical Academy of Research and Translation, Jiangxi Science and Technology Normal University, Wenzhou Institute, University of Chinese Academy of Sciences

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900

Funders: National Natural Science Foundation of China, Yunnan Provincial Science and Technology Department, National Key Research and Development Program of China, National Key Research and Development Program of China Stem Cell and Translational Research, Basic and Applied Basic Research Foundation of Guangdong Province

Awards: G1568979219, G3103666603, G363088918, G372307204, G4035405853, G5109273502, G5603992367, G6266727347, G650662272, G6888334679, G7177145545, G7462785273, G7617563361, G8117346848

OpenAlex ID: W7154631233

FWCI: 1.939

Cites: 70

Cited by: 1

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