Cell BiologybioRxiv
High-Throughput Functional Assay in Cystic Fibrosis Patient-Derived Organoids Allows Drug Repurposing
S. Spelier, E. de Poel, G. N. Ithakisiou…·J. Beekman
This study uses a high-throughput functional assay in patient-derived organoids to screen FDA-approved drugs for CFTR function restoration in rare mutations, finding statins enhance CFTR function specifically in W1282X mutations.
DOI 10.1183/23120541.00495-2022