Article

High-Throughput Functional Assay in Cystic Fibrosis Patient-Derived Organoids Allows Drug Repurposing

2022-07-15

Abstract excerpt

<h4>ABSTRACT</h4> Cystic fibrosis (CF) is a rare hereditary disease caused by mutations in the CFTR gene. Recent therapies enable effective restoration of CFTR function of the most common F508del CFTR mutation. This shifts the unmet clinical need towards people with rare CFTR mutations such as nonsense mutations, of which G542X and W1282X are most prevalent. CFTR function measurements in patient-derived cell-base...

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Literature Corpus work: 646753eb-a20c-524e-960d-207967c81be5
DOI: 10.1101/2022.07.14.500147

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