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...

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Bibliografiska uppgifter
I publikationen:Int J Mol Sci
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: MDPI 2020
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7177453/
https://ncbi.nlm.nih.gov/pubmed/32244302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21072398
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