Nucleic acid therapy for metabolic-related diseases  

作　　者：Jing Guo Zhi-Guo Lu Rui-Chen Zhao Bao-Ku Li Xin Zhang 

机构地区：[1]College of Pharmaceutical Sciences,Hebei University,Baoding 071002,China [2]State Key Laboratory of Biochemical Engineering,Institute of Process Engineering,Chinese Academy of Sciences,Beijing 100190,China [3]School of Chemical Engineering,University of Chinese Academy of Sciences,Beijing 100049,China [4]Key Laboratory of Biopharmaceutical Preparation and Delivery,Institute of Process Engineering,Chinese Academy of Sciences,Beijing 100190,China

出　　处：《Chinese Chemical Letters》2025年第3期83-94,共12页中国化学快报(英文版)

基　　金：financially supported by the National Natural Science Foundation of China(Nos.32225029,22205240,52073287,22075289,82071552 and 22376006);National Key R&D Program of China(No.2023YFC2605003)。

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 metabolismrelated 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.

关 键 词：Metabolism-related diseases Signal pathways Target genes Nucleic acid therapy Delivery technologies 

分 类 号：R58[医药卫生—内分泌] R589[医药卫生—内科学]