Cystic fibrosis: a model system for precision medicine.

ABSTRACT:

Purpose of review

Development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, small molecule therapies that target the basic defect in cystic fibrosis (CF), represents a new era in CF treatment. This review highlights recent progress in CF therapeutics as an example of precision medicine and personalized approaches to test CFTR modulators using preclinical model systems.

Recent findings

CFTR modulators are now clinically available for approximately 50% of the United States CF population. The CFTR potentiator, ivacaftor, is approved for people with CF ages 2 years and older with at least one gating mutation (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) or the R117H conductance mutation. The recent Food and Drug Administration

SUBMITTER: Martiniano SL 

PROVIDER: S-EPMC4946574 | biostudies-literature | 2016 Jun

REPOSITORIES: biostudies-literature