Oligonucleotide Therapies for Nonalcoholic Steatohepatitis

Overview

Journal Mol Ther Nucleic Acids

Publisher Cell Press

Date 2024 Apr 26

PMID 38665220

Authors

Sixu Li

Feng Xiong

Songbo Zhang

Jinghua Liu

Guangping Gao

Jun Xie

Yi Wang

Affiliations

Soon will be listed here.

Abstract

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.

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