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Epigenetics, DNA methylation, and chromatin modifying drugs.

机译:表观遗传学,DNA甲基化和染色质修饰药物。

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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.
机译:越来越多的证据表明,几种疾病和行为病理是由基因功能缺陷引起的。研究最好的例子是癌症,但其他疾病,例如自身免疫性疾病,哮喘,2型糖尿病,代谢紊乱和自闭症,则表现出异常的基因表达。基因功能可以通过DNA序列的改变或在不存在序列改变的情况下基因的表观遗传编程的改变来改变。使用表观遗传学药物,可以逆转与不同疾病状态相关的异常基因表达谱。在临床试验中已经测试了几种靶向DNA甲基化和组蛋白脱乙酰基酶的表观遗传药物。了解表观遗传机制及其组成成分在特定疾病状态下的不同作用对于开发靶向表观遗传疗法至关重要。

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