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Updated: Aug 8, 2026

Repressing Gene Transcription by Redirecting Cellular Machinery with Chemical Epigenetic Modifiers
Published on: September 20, 2018
Targeting cancer epigenetic pathways with small-molecule modulators for potential therapeutic purposes
1Department of Urology, Shengjing Hospital of China Medical University, Shenyang 110001, China.
Epigenetic therapies, targeting DNA methyltransferases (DNMTs) and histone-modifying enzymes, show promise in cancer treatment by altering gene expression. Further research is needed to address safety and clinical translation challenges for these novel epigenetic drugs.
Area of Science:
- Oncology
- Epigenetics
- Pharmacology
Background:
- Epigenetic regulators control gene expression and cellular processes crucial for cancer development.
- Epigenetic therapies offer a promising avenue to enhance existing cancer treatments, including overcoming drug resistance.
Purpose of the Study:
- To review recent advances in small-molecule epigenetic modulators for cancer treatment.
- To analyze their mechanisms, therapeutic applications, and clinical development challenges.
Main Methods:
- Review of recent pharmacological innovations in small-molecule inhibitors targeting epigenetic enzymes.
- Analysis of preclinical and early-phase clinical trial data for epigenetic agents.
- Assessment of safety, selectivity, and resistance profiles of epigenetic modulators.
Main Results:
- Small-molecule inhibitors targeting DNMTs, HATs, HDACs, and HMTs demonstrate therapeutic efficacy in preclinical and clinical settings.
- These agents reprogram epigenetic landscapes, potentiate conventional therapies, and overcome resistance.
- Gaps exist in comprehensive safety, selectivity, and resistance profile assessments.
Conclusions:
- Small-molecule epigenetic modulators hold significant therapeutic potential in oncology.
- Addressing challenges in clinical translation and understanding resistance mechanisms is crucial for optimizing next-generation epigenetic therapies.
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