Oligonucleotide therapies for nonalcoholic steatohepatitis

Sixu Li; Feng Xiong; Songbo Zhang; Jinghua Liu; Guangping Gao; Jun Xie; Yi Wang

Molecular Therapy: Nucleic Acids (Jun 2024)

Oligonucleotide therapies for nonalcoholic steatohepatitis

Sixu Li,
Feng Xiong,
Songbo Zhang,
Jinghua Liu,
Guangping Gao,
Jun Xie,
Yi Wang

Affiliations

Sixu Li: Department of Pathophysiology, West China College of Basic Medical Sciences & Forensic Medicine, Sichuan University, Chengdu 610066, China
Feng Xiong: Department of Cardiology, The Third People’s Hospital of Chengdu, Chengdu 610031, China
Songbo Zhang: Department of Breast Surgery, Sichuan Clinical Research Center for Cancer, Sichuan Cancer Hospital & Institute, Sichuan Cancer Center, Affiliated Cancer Hospital of University of Electronic Science and Technology of China, Chengdu 610041, China
Jinghua Liu: Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA
Guangping Gao: Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Viral Vector Core, University of Massachusetts Chan Medical, School, Worcester, MA 01605, USA
Jun Xie: Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA; Viral Vector Core, University of Massachusetts Chan Medical, School, Worcester, MA 01605, USA; Corresponding author: Jun Xie, PhD, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA.
Yi Wang: Department of Pathophysiology, West China College of Basic Medical Sciences & Forensic Medicine, Sichuan University, Chengdu 610066, China; Corresponding author: Yi Wang, PhD, Department of Pathophysiology, West China College of Basic Medical Sciences & Forensic Medicine, Sichuan University, Chengdu 610066, China.

Journal volume & issue: Vol. 35, no. 2
p. 102184

Abstract

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Nonalcoholic steatohepatitis (NASH) represents a severe disease subtype of nonalcoholic fatty liver disease (NAFLD) that is thought to be highly associated with systemic metabolic abnormalities. It is characterized by a series of substantial liver damage, including hepatocellular steatosis, inflammation, and fibrosis. The end stage of NASH, in some cases, may result in cirrhosis and hepatocellular carcinoma (HCC). Nowadays a large number of investigations are actively under way to test various therapeutic strategies, including emerging oligonucleotide drugs (e.g., antisense oligonucleotide, small interfering RNA, microRNA, mimic/inhibitor RNA, and small activating RNA) that have shown high potential in treating this fatal liver disease. This article systematically reviews the pathogenesis of NASH/NAFLD, the promising druggable targets proven by current studies in chemical compounds or biological drug development, and the feasibility and limitations of oligonucleotide-based therapeutic approaches under clinical or pre-clinical studies.

Published in Molecular Therapy: Nucleic Acids

ISSN: 2162-2531 (Online)
Publisher: Elsevier
Country of publisher: United States
LCC subjects: Medicine: Therapeutics. Pharmacology
Website: https://www.cell.com/molecular-therapy-family/nucleic-acids/latest-content

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Abstract

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