Systemic effects of cystic fibrosis transmembrane conductance regulator modulators on the plasma and serum proteome

Fentker, Kerstin; Kirchner, Marieluise; Ziehm, Matthias; Niquet, Sylvia; Popp, Oliver; Duerr, Julia; Schaupp, Laura; Roehmel, Jobst; Thee, Stephanie; Hämmerling, Susanne; Sommerburg, Olaf; Stahl, Mirjam; Graeber, Simon Y.; Mall, Marcus A.; Mertins, Philipp

Systemic effects of cystic fibrosis transmembrane conductance regulator modulators on the plasma and serum proteome

Tools

Preview	PDF (Publisher's Version) - Requires a PDF viewer such as GSview, Xpdf or Adobe Acrobat Reader 5MB
	Other (Supplemental Information) 5MB

Item Type:	Article
Title:	Systemic effects of cystic fibrosis transmembrane conductance regulator modulators on the plasma and serum proteome
Creators Name:	Fentker, Kerstin, Kirchner, Marieluise, Ziehm, Matthias, Niquet, Sylvia, Popp, Oliver, Duerr, Julia, Schaupp, Laura, Roehmel, Jobst, Thee, Stephanie, Hämmerling, Susanne, Sommerburg, Olaf, Stahl, Mirjam, Graeber, Simon Y., Mall, Marcus A. and Mertins, Philipp
Abstract:	Cystic fibrosis (CF) is caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel in epithelial organs leading to a complex multi-organ disease. CFTR modulator drugs improve mutant CFTR function and markedly improve clinical outcomes in people with CF (pwCF), but their broader molecular and systemic effects remain to be fully elucidated. We employed mass-spectrometry-based proteomics to compare the plasma and serum proteomes of pwCF treated with the dual combination CFTR modulator lumacaftor/ivacaftor (LUM/IVA) or the triple combination of elexacaftor/tezacaftor/ivacaftor (ELX/ TEZ/IVA). We found specific and common signatures associated with inflammation and metabolic processes. ELX/TEZ/IVA showed more consistent effects that were directed toward profiles in healthy individuals. Our findings indicate that ELX/TEZ/IVA leads to broader improvements in protein dysregulation than LUM/ IVA in pwCF. Residual proteome changes may inform future therapeutic strategies for CF. A record of this paper’s transparent peer review process is included in the supplemental information.
Source:	Cell Systems
ISSN:	2405-4712
Publisher:	Cell Press / Elsevier
Volume:	17
Page Range:	101569
Number of Pages:	1
Date:	1 April 2026
Official Publication:	https://doi.org/10.1016/j.cels.2026.101569
PubMed:	View item in PubMed

Repository Staff Only: item control page

Download Statistics

Downloads

Downloads per month over past year