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Updated: Jun 29, 2026

Repressing Gene Transcription by Redirecting Cellular Machinery with Chemical Epigenetic Modifiers
Published on: September 20, 2018
Epigenetics, DNA methylation, and chromatin modifying drugs
1Department of Pharmacology and Therapeutics, McGill University, Montréal, Quebec H3G 1Y6, Canada. mszyf@pharma.mcgill.ca
Abstract:
Evidence is emerging that several diseases and behavioral pathologies result from defects in gene function. The best-studied example is cancer, but other diseases such as autoimmune disease, asthma, type 2 diabetes, metabolic disorders, and autism display aberrant gene expression. Gene function may be altered by either a change in the sequence of the DNA or a change in epigenetic programming of a gene in the absence of a sequence change. With epigenetic drugs, it is possible to reverse aberrant gene expression profiles associated with different disease states. Several epigenetic drugs targeting DNA methylation and histone deacetylation enzymes have been tested in clinical trials. Understanding the epigenetic machinery and the differential roles of its components in specific disease states is essential for developing targeted epigenetic therapy.
Insights
Gene defects cause diseases like cancer and autism. Epigenetic drugs can reverse abnormal gene expression, offering a new path for targeted therapies.
Area of Science:
- Genetics
- Molecular Biology
- Epigenetics
Background:
- Aberrant gene expression is linked to numerous diseases, including cancer, autoimmune disorders, asthma, type 2 diabetes, metabolic disorders, and autism.
- Gene function can be altered by DNA sequence changes or epigenetic modifications without altering the DNA sequence.
Purpose of the Study:
- To highlight the role of epigenetic modifications in disease development.
- To discuss the potential of epigenetic drugs in reversing aberrant gene expression.
- To emphasize the need for understanding epigenetic machinery for targeted therapy development.
Main Methods:
- Review of emerging evidence on gene function defects and disease.
- Analysis of epigenetic mechanisms, including DNA methylation and histone deacetylation.
- Discussion of clinical trial data for epigenetic drugs.
Main Results:
- Epigenetic drugs show promise in reversing disease-associated gene expression profiles.
- Several epigenetic drugs targeting DNA methylation and histone deacetylation have undergone clinical trials.
- Understanding the epigenetic machinery is crucial for developing effective epigenetic therapies.
Conclusions:
- Epigenetic alterations play a significant role in various diseases.
- Targeted epigenetic therapies offer a potential treatment strategy for a range of conditions.
- Further research into epigenetic mechanisms is essential for advancing therapeutic development.
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