Developmental control of CFTR: from bioinformatics to novel therapeutic approaches.

Celebrating the 25th anniversary of identifying the genetic cause of cystic fibrosis (CF), tremendous progress has been made in the understanding and the treatment of this complex, but still fatal disease [1]. Despite advances, several key questions about the pathomechanisms of CF remain poorly understood. For instance, the precise genetic and epigenetic mechanisms that regulate expression and stability of the cystic fibrosis transmembrane conductance regulator (CFTR) gene remain incompletely defined.