Nucleic acid therapy for metabolic-related diseases

Jing Guo,Zhi-Guo Lu,Rui-Chen Zhao, Bao-Ku Li,Xin Zhang

Chinese Chemical Letters(2024)

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摘要
Metabolism is a general term for a series of ordered chemical reactions in an organism used to maintain life, mainly divided into anabolic and catabolic metabolism. Nucleic acid therapy can not only precisely up-regulate and down-regulate the expression of target genes but also correct mutated disease-causing genes, which demonstrates irreplaceable and outstanding advantages in the treatment of metabolism-related diseases and has been applied to the clinical treatment of metabolism-related diseases. In this review, we introduce the structures of several major nucleic acid drugs and the mechanism of nucleic acid therapy. Subsequently, we describe the mechanisms of various biomolecular and tissue metabolisms and the etiology of metabolic disorders, classified according to metabolic substrates. We analyze the signal pathways and potential targets affecting the metabolism of each substrate and describe the nucleic acid drugs applied to these targets and their delivery technologies. This review aims to provide new ideas and targets for treating these diseases by investigating the role played by metabolism in developing diseases and providing guidance for the selection and design of nucleic acid drugs.
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关键词
Metabolism-related diseases,Signal pathways,Target genes,Nucleic acid therapy,Delivery technologies
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