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Epigenetics is the study of changes in gene expression that do not arise from alterations in the DNA sequence, resulting in the heritable silencing of genes without changing the coding sequence. Epigenetic therapy involves using drugs or other techniques to influence these epigenetic mechanisms in addressing specific medical conditions.
A hypomethylating agent (or demethylating agent [1]) is a drug that inhibits DNA methylation: the modification of DNA nucleotides by addition of a methyl group.Because DNA methylation affects cellular function through successive generations of cells without changing the underlying DNA sequence, treatment with a hypomethylating agent is considered a type of epigenetic therapy.
Individual differences in drug metabolism and response can be partially explained by epigenetic changes. [7] [8] Epigenetic changes in genes that encode drug targets, enzymes, or transport proteins that affect the body's ability to absorb, metabolize, distribute and excrete substances that are foreign to the body (Xenobiotics) can result in changes in one's toxicity levels and drug response.
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
BET inhibitors have been developed by publicly funded research labs as well as pharmaceutical companies including GlaxoSmithKline, Oncoethix (purchased by Merck & Co. in 2014 [20]), Oncoethix, [21] Constellation pharmaceuticals, [22] Resverlogix Corp [23] and Zenith epigenetics. [24]
After a lull of nearly 2 decades, the Food and Drug Administration (FDA) has approved some novel drugs for the treatment of Alzheimer’s disease since 2021. Most of these drugs are antibody ...
To carry out gene expression, a cell must control the coiling and uncoiling of DNA around histones.This is accomplished with the assistance of histone acetyl transferases (HAT), which acetylate the lysine residues in core histones leading to a less compact and more transcriptionally active euchromatin, and, on the converse, the actions of histone deacetylases (HDAC), which remove the acetyl ...