High-Throughput Functional Assay in Cystic Fibrosis Patient-Derived Organoids Allows Drug Repurposing
S. Spelier, E. de Poel, G. N. Ithakisiou, S. Suen, M. Hagemeijer, D. Muijlwijk, A. Vonk, J. Brunsveld, E. Kruisselbrink, K. van der Ent, 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.
bioRxiv · Jul 14, 2022Cell Biology