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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...
| Autores principales: | , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2021
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| Materias: | |
| 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 |