BioCentury
ARTICLE | Preclinical News

Inhibiting the ubiquitin ligase RNF5 to treat CF

May 11, 2018 10:15 PM UTC

In a paper published in Cell Chemical Biology, Italian researchers identified a pharmacological inhibitor of ring finger protein 5 (RNF5) that prevented the premature degradation of mutant F508del cystic fibrosis transmembrane conductance regulator (CFTR) protein, providing evidence of the "druggability" of the E3 ubiquitin ligase as a target for CF therapies.

The F508del mutation occurs in about 90% of all mutated CFTR alleles and causes CFTR misfolding that leads to decreased CFTR activity and reduced expression of the protein on the cell surface...