Treatment of Cystic Fibrosis Patients Homozygous for <i>F508del</i> with Lumacaftor-Ivacaftor (Orkambi<sup>®</sup>) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells

Treatment of Cystic Fibrosis Patients Homozygous for <i>F508del</i> with Lumacaftor-Ivacaftor (Orkambi<sup>®</sup>) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells

The treatment of cystic fibrosis (CF) patients homozygous for the <i>F508del</i> mutation with Orkambi<sup>®</sup>, a combination of a corrector (lumacaftor) and a potentiator (ivacaftor) of the mutated CFTR protein, resulted in some amelioration of the respiratory function....

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Bibliographic Details
Main Authors: Maria Favia, Crescenzio Gallo, Lorenzo Guerra, Domenica De Venuto, Anna Diana, Angela Maria Polizzi, Pasqualina Montemurro, Maria Addolorata Mariggiò, Giuseppina Leonetti, Antonio Manca, Valeria Casavola, Massimo Conese
Format: Article
Language:English
Published: MDPI AG 2020-03-01
Series:International Journal of Molecular Sciences
Subjects:
cystic fibrosis
Orkambi<sup>®</sup>
CFTR
mononuclear cells
BMI
sweat chloride
Online Access:https://www.mdpi.com/1422-0067/21/7/2398

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