Article
Cystic fibrosis: a model system for precision medicine.
Current opinion in pediatrics - 1 Jun 2016
Martiniano Stacey L, Sagel Scott D, Zemanick Edith T
Abstract excerpt
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...
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