Title:Epigenetics in Cystic Fibrosis: Epigenetic Targeting of a Genetic Disease
Volume: 16
Issue: 9
Author(s): Nualpun Sirinupong and Zhe Yang
Affiliation:
Keywords:
CFTR, cystic fibrosis, DNA methylation, epigenetics, histone acetylation, histone methylation.
Abstract: 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.
