Bone demineralization is improved by ivacaftor in patients with cystic fibrosis carrying the p.Gly551Asp mutation - Archive ouverte HAL Access content directly
Journal Articles Journal of Cystic Fibrosis Year : 2016

Bone demineralization is improved by ivacaftor in patients with cystic fibrosis carrying the p.Gly551Asp mutation

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Abstract

Low bone mineral density (BMD) is a common problem in adults with cystic fibrosis (CF), the etiology of which is multifactorial. In this study, we provide the first evidence that ivacaftor improves BMD in CF patients carrying the p.Gly551Asp mutation. Consistently, in vitro experiments with TNF-α-stimulated primary F508del-CFTR osteoblasts demonstrated that correction of p.Phe508del-CFTR markedly decreased RANKL protein production, a major factor of bone resorption. These clinical and fundamental observations suggest that rescue of mutated CFTR protein improves bone remodeling and support the link between CFTR and bone cell physiology. These findings represent a step forward in the development of potential new therapies for CF-related bone disease.

Dates and versions

hal-03475342 , version 1 (10-12-2021)

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Isabelle Sermet-Gaudelus, Martial Delion, Isabelle Durieu, Jacky Jacquot, Dominique Hubert. Bone demineralization is improved by ivacaftor in patients with cystic fibrosis carrying the p.Gly551Asp mutation. Journal of Cystic Fibrosis, 2016, 15, pp.e67 - e69. ⟨10.1016/j.jcf.2016.09.003⟩. ⟨hal-03475342⟩
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