Article
Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype.
Molecular genetics & genomic medicine - 1 Apr 2021
Terlizzi Vito, Amato Felice, Castellani Chiara, Ferrari Beatrice, Galietta Luis J V, Castaldo Giuseppe, Taccetti Giovanni
Abstract excerpt
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 uncharacterized in vitro and that are not included in clinical trials. For this purpose, ex vivo models are necessary to...
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