Whole-blood transcriptomic responses to lumacaftor/ivacaftor therapy in cystic fibrosis

Cystic fibrosis (CF) remains without a definitive cure. Novel therapeutics targeting the causative defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene are in clinical use. Lumacaftor/ivacaftor is a CFTR modulator approved for patients homozygous for the CFTR variant p.Phe508del, but there are wide variations in treatment responses preventing prediction of patient responses. We aimed to determine changes in gene expression related to treatment initiation and response.

Source: Journal of Cystic Fibrosis - August 28, 2019 Category: Respiratory Medicine Authors: Benjamin T. Kopp, James Fitch, Lisa Jaramillo, Chandra L. Shrestha, Frank Robledo-Avila, Shuzhong Zhang, Sabrina Palacios, Fred Woodley, Don Hayes, Santiago Partida-Sanchez, Octavio Ramilo, Peter White, Asuncion Mejias Source Type: research