Tingyi Li,1,2 Yanwei Chen,1 Shuai Li1 

1Department of Pharmacy, The First Affiliated Hospital of Dalian Medical University, Dalian, People’s Republic of China; 2Dalian Medical University, Dalian, People’s Republic of China

Correspondence: Shuai Li; Yanwei Chen, Department of Pharmacy, The First Affiliated Hospital of Dalian Medical University, Dalian, People’s Republic of China, Email shuaili2015@outlook.com; chenyanweile@163.com

Abstract: Epigenetic dysregulation can significantly trigger the onset and progression of various diseases, epigenetic therapy is a new treatment strategy by changing DNA methylation, histone modification, N6-methyladenosine, chromatin modification and other epigenetic modifications to regulate gene expression levels for therapeutic purposes. However, small-molecule epigenetic drugs face challenges in disease treatment, such as lack of selectivity, limited therapeutic efficacy, and insufficient safety. Nanomedicine delivery systems offer significant advantages in addressing these issues by enhancing drug targeting, improving bioavailability, and reducing nonspecific distribution. This help minimize side effects while increasing both therapeutic effectiveness and safety of epigenetic drugs. In this review, we focus on the mechanism and role of epigenetic regulatory factors in diseases, as well as the challenges faced by small molecule inhibitors in treatment strategies, especially the research advancements in epigenetic drug delivery systems, review and discuss the therapeutic potential and challenges of using nanotechnology to develop epigenetic drug delivery systems.

Keywords: epigenetic therapy, DNA methylation, N6-methyladenosine, histone modification, nanomedicine delivery systems, nanotechnology