PT - JOURNAL ARTICLE AU - Daniel H. Tewkesbury AU - Rebecca C. Robey AU - Peter J. Barry TI - Progress in precision medicine in cystic fibrosis: a focus on CFTR modulator therapy AID - 10.1183/20734735.0112-2021 DP - 2021 Dec 01 TA - Breathe PG - 210112 VI - 17 IP - 4 4099 - http://breathe.ersjournals.com/content/17/4/210112.short 4100 - http://breathe.ersjournals.com/content/17/4/210112.full SO - Breathe2021 Dec 01; 17 AB - The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in therapeutic approaches within the past decade. Since the first clinical descriptions in the 1930s, treatment advances had focused on the downstream consequences of a dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) chloride ion channel. The discovery of the gene that codes for CFTR and an understanding of the way in which different genetic mutations lead to disruption of normal CFTR function have led to the creation and subsequent licensing of drugs that target this process. This marks an important move towards precision medicine in CF and results from clinical trials and real-world clinical practice have been impressive. In this review we outline how CFTR modulator drugs restore function to the CFTR protein and the progress that is being made in this field. We also describe the real-world impact of CFTR modulators on both pulmonary and multisystem complications of CF and what this will mean for the future of CF care.CFTR modulator drugs target the underlying cellular defect in cystic fibrosis. They have a range of pulmonary and multisystem effects and are leading to a transformation of care for a majority of people with cystic fibrosis. https://bit.ly/3nfEt8i