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

The treatment of cystic fibrosis (CF) patients homozygous for the F508del mutation with Orkambi(®), a combination of a corrector (lumacaftor) and a potentiator (ivacaftor) of the mutated CFTR protein, resulted in some amelioration of the respiratory function. However, a great variability in the clin...

Descripción completa

Detalles Bibliográficos
Autores principales: Favia, Maria, Gallo, Crescenzio, Guerra, Lorenzo, De Venuto, Domenica, Diana, Anna, Polizzi, Angela Maria, Montemurro, Pasqualina, Mariggiò, Maria Addolorata, Leonetti, Giuseppina, Manca, Antonio, Casavola, Valeria, Conese, Massimo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7177453/
https://www.ncbi.nlm.nih.gov/pubmed/32244302
http://dx.doi.org/10.3390/ijms21072398

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7177453/
https://www.ncbi.nlm.nih.gov/pubmed/32244302
http://dx.doi.org/10.3390/ijms21072398

Ejemplares similares