High-throughput functional assay in cystic fibrosis patient-derived organoids allows drug repurposing

BACKGROUND: Cystic fibrosis (CF) is a rare hereditary disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Recent therapies enable effective restoration of CFTR function of the most common F508del CFTR mutation. This shifts the unmet clinical need towar...

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Detalles Bibliográficos
Autores principales: Spelier, Sacha, de Poel, Eyleen, Ithakisiou, Georgia N., Suen, Sylvia W.F., Hagemeijer, Marne C., Muilwijk, Danya, Vonk, Annelotte M., Brunsveld, Jesse E., Kruisselbrink, Evelien, van der Ent, Cornelis K., Beekman, Jeffrey M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: European Respiratory Society 2023
Materias:
Original Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9885274/
https://www.ncbi.nlm.nih.gov/pubmed/36726369
http://dx.doi.org/10.1183/23120541.00495-2022

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