A bioassay using intestinal organoids to measure CFTR modulators in human plasma.

Autor: Dekkers R; Laboratory of Translational Immunology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands., Vijftigschild LA; Laboratory of Translational Immunology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands., Vonk AM; Laboratory of Translational Immunology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands., Kruisselbrink E; Laboratory of Translational Immunology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands., de Winter-de Groot KM; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands., Janssens HM; Department of Pediatric Pulmonology, Erasmus University Medical Center/Sophia Children's Hospital, Rotterdam, The Netherlands., van der Ent CK; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands., Beekman JM; Laboratory of Translational Immunology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands; Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands. Electronic address: JBeekman@umcutrecht.nl.
Jazyk: angličtina
Zdroj: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2015 Mar; Vol. 14 (2), pp. 178-81. Date of Electronic Publication: 2014 Nov 06.
DOI: 10.1016/j.jcf.2014.10.007
Abstrakt: Treatment efficacies of drugs depend on patient-specific pharmacokinetic and pharmacodynamic properties. Here, we developed an assay to measure functional levels of the CFTR potentiator VX-770 in human plasma and observed that VX-770 in plasma from different donors induced variable CFTR function in intestinal organoids. This assay can help to understand variability in treatment response to CFTR potentiators by functionally modeling individual pharmacokinetics.
(Copyright © 2014. Published by Elsevier B.V.)
Databáze: MEDLINE
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