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.
