The Evolution of Precision Medicine in Cystic Fibrosis

Cystic fibrosis (CF) is the most common fatal genetic disease in the US. Since the sequencing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989, better characterization of CFTR mutations led CF to become the first lung disease applying personalized medicine for diagnostic and therapeutic purposes. Advances in CFTR modulators, gene therapy, and new drug efficacy screening methods are creating individualized treatment opportunities the majority of CF patients regardless of their CFTR genotype. These investigative efforts will define novel and personalized therapeutic targets that minimize the impact of CF on the health and quality of life of our patients.