Work

Effect of elexacaftor and bamocaftor on the metabolic and thermal stability of the F508del-CFTR protein in human airway epithelial cells

Year: 2025

Type: article

Abstract: Our in vitro study underscores that ETI/BTI's efficacy in improving F508del-CFTR function depends on treatment concentration and duration, impacting the protein's metabolic and thermal stability. Although ETI/BTI only partially addresses F508del-CFTR's inherent thermal instability, reduced doses retained significant effectiveness. This finding supports dose optimization as a promising strategy to sustain therapeutic benefits while minimizing side effects, offering a personalized approach to treatment for individuals with cystic fibrosis experiencing adverse effects from standard dosing.

Source: American Journal of Physiology-Lung Cellular and Molecular Physiology

DOI: 10.1152/ajplung.00011.2025

Authors: Thomas Carrez, Sandra Mirval, Christine Barrault, Florian Devetter, Clarisse Vandebrouck, Frédéric Becq

Institution: Université de Poitiers

Language: English

Topic: Cystic Fibrosis Research Advances

Subfield: Pulmonary and Respiratory Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Funders: Association Nationale de la Recherche et de la Technologie, Association Vaincre la Mucoviscidose