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FDA-Approved Drug Screening in Patient-Derived Organoids Demonstrates Potential of Drug Repurposing for Rare Cystic Fibrosis Genotypes

2022-09-03

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Preclinical cell-based assays that recapitulate human disease play an important role in drug repurposing. We previously developed a functional forskolin induced swelling (FIS) assay using patient-derived intestinal organoids (PDIOs), allowing functional characterization of CFTR, the gene mutated in people with cystic fibrosis (pwCF). CFTR function-increasing pharmacotherapie...

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Literature Corpus work: ae82ad15-23a9-5bc7-9ce8-37d27e1b340f
DOI: 10.1101/2022.09.02.506304

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