Article
ORIGIN-1 trial: study protocol for an organoid-guided N-of-1 trial evaluating CFTR modulator response in cystic fibrosis with rare non-F508del mutations.
Trials - 15 Jun 2026
Sabino Lorena Rodrigues, Kaiko Gerard E, Dorahy Douglas, Goodsall Thomas, Jaffe Adam, Schultz Andre, Waters Shafagh, Cook Dane, Kok Harvey, Tong Koliarne, Totterdell James, McGree James, Snelling Tom, Wark Peter A B
Abstract excerpt
BACKGROUND: Cystic fibrosis (CF) is a genetic condition caused by mutation of the cystic fibrosis transmembrane regulator (CFTR) gene. Recently licensed modulator therapies target the defective CFTR protein and have transformed the formerly life-limiting trajectory of people with CF (pwCF). elexacaftor/tezacaftor/ivacaftor (ETI) has shown outstanding clinical efficacy in pwCF homozygous for F508del (approximately...
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