ETABLISSEMENT FRANÇAIS DU SANG (EFS);UNIVERSITÉ DE BRETAGNE OCCIDENTALE;CENTRE HOSPITALIER REGIONAL ET UNIVERSITAIRE DE BREST;INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE)
Activating transcription factor 6 (ATF6) is involved in cystic fibrosis transmembrane conductance regulator (CFTR) repression and understanding this inhibitory mechanism is of great interest to develop a therapeutic approach based on UPR regulation. Using site-1 protease (S1P) inhibitor (e.g. PF-429242) the inventors showed that both membrane localization and function of F508del-CFTR are partially restored. Accordingly, the present invention relates to a method of treating a disease associated with reduced CFTR function in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a S1P inhibitor.
