Use of guanabenz compounds to prepare medicaments for treating cystic fibrosis and diseases associated with protein addressing deficiency in cells

摘要

Guanabenz compounds (I) are used to prepare medicaments for treating cystic fibrosis and diseases associated with protein addressing deficiency in cells. Guanabenz compounds of formula Ar-CH=N-NH-C(NH)-NH 2(I) or their salts are used to prepare medicaments for treating cystic fibrosis and diseases associated with protein addressing deficiency. Ar : phenyl with at least two chloro substituents. ACTIVITY : CNS-Gen.; Respiratory-Gen. MECHANISM OF ACTION : Cystic fibrosis transmembrane conductance regulator (CFTR) activator. Iodide efflux experiments performed on CF15 human pulmonary epithelial cells, which are homozygotic for the CFTR protein deletion mutant delF508, showed that guanabenz promoted relocation of del508-CFTR in the cell membrane after 2 hours with an EC 50of 40 mu M.