Article
Targeted Therapy for Restoring CFTR Activity: From Experimental to Clinical Features.
International journal of molecular sciences - 1 Aug 2026
Allushi Sara, Virgulti Mariarita, Blaconà Giovanna, Ferraguti Giampiero, Eramo Adriana, Lucarelli Marco
Abstract excerpt
Cystic fibrosis (CF) is one of the most common rare genetic diseases. It is caused by pathogenic variants of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. More than 2200 variants have been identified in the CFTR gene that need detailed knowledge and functional characterization in order to develop specific therapeutic strategies. The traditional therapy for CF relied on addressing symptoms...
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