Epigenetics-targeted drugs: current paradigms and future challenges

Abstract Epigenetics governs a chromatin state regulatory system through five key mechanisms: DNA modification, histone modification, RNA modification, chromatin remodeling, and non-coding RNA regulation. These mechanisms and their associated enzymes convey genetic information independently of DNA b...

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Main Authors: Wanlin Dai, Xinbo Qiao, Yuanyuan Fang, Renhao Guo, Peng Bai, Shuang Liu, Tingting Li, Yutao Jiang, Shuang Wei, Zhijing Na, Xue Xiao, Da Li
Format: Article
Language:English
Published: Nature Publishing Group 2024-11-01
Series:Signal Transduction and Targeted Therapy
Online Access:https://doi.org/10.1038/s41392-024-02039-0
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author Wanlin Dai
Xinbo Qiao
Yuanyuan Fang
Renhao Guo
Peng Bai
Shuang Liu
Tingting Li
Yutao Jiang
Shuang Wei
Zhijing Na
Xue Xiao
Da Li
author_facet Wanlin Dai
Xinbo Qiao
Yuanyuan Fang
Renhao Guo
Peng Bai
Shuang Liu
Tingting Li
Yutao Jiang
Shuang Wei
Zhijing Na
Xue Xiao
Da Li
author_sort Wanlin Dai
collection DOAJ
description Abstract Epigenetics governs a chromatin state regulatory system through five key mechanisms: DNA modification, histone modification, RNA modification, chromatin remodeling, and non-coding RNA regulation. These mechanisms and their associated enzymes convey genetic information independently of DNA base sequences, playing essential roles in organismal development and homeostasis. Conversely, disruptions in epigenetic landscapes critically influence the pathogenesis of various human diseases. This understanding has laid a robust theoretical groundwork for developing drugs that target epigenetics-modifying enzymes in pathological conditions. Over the past two decades, a growing array of small molecule drugs targeting epigenetic enzymes such as DNA methyltransferase, histone deacetylase, isocitrate dehydrogenase, and enhancer of zeste homolog 2, have been thoroughly investigated and implemented as therapeutic options, particularly in oncology. Additionally, numerous epigenetics-targeted drugs are undergoing clinical trials, offering promising prospects for clinical benefits. This review delineates the roles of epigenetics in physiological and pathological contexts and underscores pioneering studies on the discovery and clinical implementation of epigenetics-targeted drugs. These include inhibitors, agonists, degraders, and multitarget agents, aiming to identify practical challenges and promising avenues for future research. Ultimately, this review aims to deepen the understanding of epigenetics-oriented therapeutic strategies and their further application in clinical settings.
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publishDate 2024-11-01
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spelling doaj-art-5b9b353abd20409c96c6fd26f07a5dba2024-12-01T12:47:04ZengNature Publishing GroupSignal Transduction and Targeted Therapy2059-36352024-11-019117110.1038/s41392-024-02039-0Epigenetics-targeted drugs: current paradigms and future challengesWanlin Dai0Xinbo Qiao1Yuanyuan Fang2Renhao Guo3Peng Bai4Shuang Liu5Tingting Li6Yutao Jiang7Shuang Wei8Zhijing Na9Xue Xiao10Da Li11Center of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityDepartment of Orthopedics, Shengjing Hospital of China Medical UniversityCenter of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityCenter of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityDepartment of Forensic Genetics, West China School of Basic Medical Sciences & Forensic Medicine, Sichuan UniversityShenyang Maternity and Child Health HospitalDepartment of General Internal Medicine VIP Ward, Liaoning Cancer Hospital & InstituteCenter of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityCenter of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityCenter of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityDepartment of Gynecology and Obstetrics, West China Second Hospital, Sichuan UniversityCenter of Reproductive Medicine, Department of Obstetrics and Gynecology, Shengjing Hospital of China Medical UniversityAbstract Epigenetics governs a chromatin state regulatory system through five key mechanisms: DNA modification, histone modification, RNA modification, chromatin remodeling, and non-coding RNA regulation. These mechanisms and their associated enzymes convey genetic information independently of DNA base sequences, playing essential roles in organismal development and homeostasis. Conversely, disruptions in epigenetic landscapes critically influence the pathogenesis of various human diseases. This understanding has laid a robust theoretical groundwork for developing drugs that target epigenetics-modifying enzymes in pathological conditions. Over the past two decades, a growing array of small molecule drugs targeting epigenetic enzymes such as DNA methyltransferase, histone deacetylase, isocitrate dehydrogenase, and enhancer of zeste homolog 2, have been thoroughly investigated and implemented as therapeutic options, particularly in oncology. Additionally, numerous epigenetics-targeted drugs are undergoing clinical trials, offering promising prospects for clinical benefits. This review delineates the roles of epigenetics in physiological and pathological contexts and underscores pioneering studies on the discovery and clinical implementation of epigenetics-targeted drugs. These include inhibitors, agonists, degraders, and multitarget agents, aiming to identify practical challenges and promising avenues for future research. Ultimately, this review aims to deepen the understanding of epigenetics-oriented therapeutic strategies and their further application in clinical settings.https://doi.org/10.1038/s41392-024-02039-0
spellingShingle Wanlin Dai
Xinbo Qiao
Yuanyuan Fang
Renhao Guo
Peng Bai
Shuang Liu
Tingting Li
Yutao Jiang
Shuang Wei
Zhijing Na
Xue Xiao
Da Li
Epigenetics-targeted drugs: current paradigms and future challenges
Signal Transduction and Targeted Therapy
title Epigenetics-targeted drugs: current paradigms and future challenges
title_full Epigenetics-targeted drugs: current paradigms and future challenges
title_fullStr Epigenetics-targeted drugs: current paradigms and future challenges
title_full_unstemmed Epigenetics-targeted drugs: current paradigms and future challenges
title_short Epigenetics-targeted drugs: current paradigms and future challenges
title_sort epigenetics targeted drugs current paradigms and future challenges
url https://doi.org/10.1038/s41392-024-02039-0
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