Rationale and design of the HIT-CF organoid study: stratifying cystic fibrosis patients based on intestinal organoid response to different CFTR-modulators

• Published in: Translational medicine communications Vol. 5; no. 1; pp. 1 - 8
• Main Authors: van Mourik, Peter, Michel, Sabine, Vonk, Annelotte M., Beekman, Jeffrey M., van der Ent, Cornelis K., De Keyser, Hilde, Lammertyn, Elise, van Koningsbruggen-Rietschel, Silke, Naehrlich, Lutz, Pool, Judith, van de Craen, Marc, Aguilera, Begoña, Pott, Johanna, Vries, Rob G. J., Boj, Sylvia F., De Boeck, Kris, Vermeulen, François, Ramalho, Anabela S., Silva, Iris A. L., Amaral, Margarida D., Goddeeris, Matthew, Gilmartin, Geoffrey
• Format: Journal Article
• Language: English
• Published: London BioMed Central 03.06.2020; BMC
• Subjects: Biobanks; Biopsy; CFTR-modulators; Clinical trials; Cystic fibrosis; Drug screening; Drugs; Laboratories; Mutation; Organoids; Patients; Personalized medicine; Pharmaceutical industry; Proteins; Regulatory approval; Reimbursement; Europe

Abstract Background Cystic fibrosis is a rare recessive monogenic disease caused by loss-of-function mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Recently developed CFTR-modulators (i.e. drugs that rescue CFTR-function) have improved the prognosis of the disease,...