Rectal organoid-guided CFTR modulator therapy restores lung function in a cystic fibrosis patient with the rare 1677delTA/R334W genotype

Ex vivo analysis of intestinal organoids has great translational potential as a precision medicine tool for the prediction of clinical efficacy of CFTR modulators and, for patients with rare variants, may hold the key to these life-changing therapies http://bit.ly/3G1FkDp