Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …

Many diseases are driven by proteins that are aberrantly ubiquitinated and degraded. These
diseases would be therapeutically benefited by targeted protein stabilization (TPS). Here we …

Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous …

Deletion of phenylalanine at position 508 (F508del) in the CFTR chloride channel is the
most frequent mutation in cystic fibrosis (CF) patients. F508del impairs the stability and …

In cystic fibrosis (CF), deletion of phenylalanine 508 (F508del) in the CF transmembrane
conductance regulator (CFTR) is associated to misfolding and defective gating of the mutant …

Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR
gene. The 3272–26A> G and 3849+ 10kbC> T CFTR mutations alter the correct splicing of …

The expansion of the CRISPR-Cas toolbox is highly needed to accelerate the development
of therapies for genetic diseases. Here, through the interrogation of a massively expanded …

Background Cystic fibrosis is caused by mutations impairing expression, trafficking, stability
and/or activity of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride …

In cystic fibrosis (CF), the deletion of phenylalanine 508 (F508del) in the CF transmembrane
conductance regulator (CFTR) leads to misfolding and premature degradation of the mutant …

Loss-of-function mutations of the CFTR gene cause cystic fibrosis (CF) through a variety of
molecular mechanisms involving altered expression, trafficking, and/or activity of the CFTR …