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Updated: Jun 29, 2026
10:28
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
Annual Review of Pharmacology and Toxicology
|October 15, 2008
Summary
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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01:37Epigenetic Regulation
Epigenetic changes alter the physical structure of the DNA without changing the genetic sequence and often regulate whether genes are turned on or off. This regulation ensures that each cell produces only proteins necessary for its function. For example, proteins that promote bone growth are not produced in muscle cells. Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
X-chromosome...
X-chromosome...
01:46Epigenetic Regulation
Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
01:46Epigenetic Regulation
Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
02:32Histone Modification
The histone proteins have a flexible N-terminal tail extending out from the nucleosome. These histone tails are often subjected to post-translational modifications such as acetylation, methylation, phosphorylation, and ubiquitination. Particular combinations of these modifications form “histone codes” that influence the chromatin folding and tissue-specific gene expression.
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