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...

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Detalles Bibliográficos
Autores principales: Terlizzi, Vito, Amato, Felice, Castellani, Chiara, Ferrari, Beatrice, Galietta, Luis J. V., Castaldo, Giuseppe, Taccetti, Giovanni
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Clinical Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8123755/
https://www.ncbi.nlm.nih.gov/pubmed/33713579
http://dx.doi.org/10.1002/mgg3.1656

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8123755/
https://www.ncbi.nlm.nih.gov/pubmed/33713579
http://dx.doi.org/10.1002/mgg3.1656

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