News

High Variability Found in People With F508del/5T;TG12 Genotype

There is wide clinical variety in the presentation of individuals who carry one F508del mutation and one 5T;TG12 mutation in the CFTR gene, an Italian study highlights. Its researchers stressed the need to monitor individuals with this genotype, since there are not currently accurate ways to predict who is at highest risk…

Aerosolized Therapy Restores CFTR in Patient-derived Cells

ReCode Therapeutics’ investigational aerosolized RNA-based therapies for cystic fibrosis (CF) restored CFTR function, the defective protein in CF, in patient-derived lung cells. Delivered by the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform, the therapy showed no signs of toxicity in epithelial cells that line the airways.

Changes in Airway Metabolites May Predict Likelihood of NTM Infection

Cystic fibrosis (CF) patients with non-tuberculous mycobacteria (NTM) lung infection show significant changes in airway metabolites relative to those without such infections, a study shows. Metabolites are intermediate or end products of cellular metabolism, and some of these altered metabolites play roles in immune responses and bacterial growth. Combining…