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

BACKGROUND: New drugs that target the basic defect in cystic fibrosis (CF) patients may now be used in a large number of patients carrying responsive mutations. Nevertheless, further research is needed to extend the benefit of these treatments to patients with rare mutations that are still uncharact...

Full description

Saved in:
Bibliographic Details
Published in:Mol Genet Genomic Med
Main Authors: Terlizzi, Vito, Amato, Felice, Castellani, Chiara, Ferrari, Beatrice, Galietta, Luis J. V., Castaldo, Giuseppe, Taccetti, Giovanni
Format: Artigo
Language:Inglês
Published: John Wiley and Sons Inc. 2021
Subjects:
Clinical Reports
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC8123755/
https://ncbi.nlm.nih.gov/pubmed/33713579
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mgg3.1656
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items