inh-02 (RNF5 inhibitor inh-02) is a novel small molecule inhibitor of E3 ubiquitin ligase RNF5/RMA1.
inh-02 causes significant F508del-CFTR rescue (EC50=2.2 uM) in immortalized and primary bronchial epithelial cells from CF patients homozygous for the F508del mutation.
inh-02 decreases ubiquitylation of mutant CFTR, causing stabilization of the mature form of CFTR.
inh-02 acts on ATG4B and paxillin, which are known targets downstream of RNF5.
