Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype.

Vito TerlizziFelice AmatoChiara CastellaniBeatrice FerrariLuis J V GaliettaGiuseppe CastaldoGiovanni Taccetti

Published in: Molecular genetics & genomic medicine (2021)

Drug response obtained on nasal epithelial cells correlates with changes in vivo therapeutic endpoints and can be a predictor of clinical efficacy of novel drugs especially in pediatric patients.

Keyphrases

cystic fibrosis
mental health
drug induced
chronic rhinosinusitis
adverse drug
stem cells
mesenchymal stem cells
cell therapy
smoking cessation