摘要
囊性纤维化(CF)是一种致命的遗传性疾病,影响肺和消化系统。CF跨膜传导调节蛋白(CFTR)基因突变是引起的疾病原因。表观遗传学如何调控CFTR表达仍然知之甚少。表观遗传学是一种机制,它是改变基因表达而不改变基本的DNA序列。表观遗传机制包括DNA甲基化和组蛋白修饰。以上两种机制都与CFTR基因的调控有关。在这里,我们综述了CFTR基因转录的表观遗传调控,讨论了潜在的表观遗传靶向策略,包括DNA甲基化,组蛋白去乙酰化酶和组蛋白甲基转移酶和去甲基化酶的抑制作用。由于表观遗传学的可逆性,靶向表观遗传机制已经是一个有吸引力的治疗方法。然而,表观遗传靶向治疗CF现仍处于其婴儿期。
关键词: 囊性纤维化跨膜电导调节因子(CFTR),囊性纤维化,DNA甲基化,表观遗传学,组蛋白乙酰化,组蛋白甲基化
图形摘要
Current Drug Targets
Title:Epigenetics in Cystic Fibrosis: Epigenetic Targeting of a Genetic Disease
Volume: 16 Issue: 9
Author(s): Nualpun Sirinupong and Zhe Yang
Affiliation:
关键词: 囊性纤维化跨膜电导调节因子(CFTR),囊性纤维化,DNA甲基化,表观遗传学,组蛋白乙酰化,组蛋白甲基化
摘要: Cystic fibrosis (CF) is a deadly genetic disease that affects the lungs and digestive system. A mutation in the CF transmembrane conductance regulator (CFTR) gene is the cause of the disease. How epigenetics contributes to CFTR expression is still poorly understood. Epigenetics is a mechanism that alters gene expression without changing the underlying DNA sequence. Epigenetic mechanisms include DNA methylation and histone modification. Both mechanisms have been implicated in CFTR gene regulation. Here we review epigenetic regulation of CFTR transcription while discussing potential epigenetic targeting strategies including DNA methyltransferase, histone deacetylase, and histone methyltransferase and demethylase inhibition. Because of the reversibility of epigenetics, targeting epigenetic mechanisms has been an attractive therapeutic approach. However, epigenetic targeting of CF disease is still at its infant stage.
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Cite this article as:
Nualpun Sirinupong and Zhe Yang , Epigenetics in Cystic Fibrosis: Epigenetic Targeting of a Genetic Disease, Current Drug Targets 2015; 16 (9) . https://dx.doi.org/10.2174/1389450116666150416114514
DOI https://dx.doi.org/10.2174/1389450116666150416114514 |
Print ISSN 1389-4501 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-5592 |
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