CFTR Rescue by Lumacaftor (VX-809) Induces an Extensive Reorganization of Mitochondria in the Cystic Fibrosis Bronchial Epithelium.
Clarissa BracciaJosie A ChristopherOliver M CrookLisa M BreckelsRayner M L QueirozNara LiessiValeria TomatiValeria CapurroTiziano BandieraSimona BaldassariNicoletta PedemonteKathryn S LilleyAndrea ArmirottiPublished in: Cells (2022)
We observed that treatment with VX-809 induces extensive structural and functional remodelling of mitochondria and peroxisomes that resemble the phenotype of healthy cells. Our data suggest additional rescue mechanisms of VX-809 beyond the correction of aberrant folding of F508del-CFTR and subsequent trafficking to the PM.
Keyphrases
- cystic fibrosis
- pseudomonas aeruginosa
- lung function
- induced apoptosis
- cell death
- cell cycle arrest
- reactive oxygen species
- air pollution
- endoplasmic reticulum
- particulate matter
- single molecule
- big data
- signaling pathway
- endoplasmic reticulum stress
- oxidative stress
- replacement therapy
- combination therapy
- data analysis
- water soluble