Powered by OpenAIRE graph
Found an issue? Give us feedback
image/svg+xml art designer at PLoS, modified by Wikipedia users Nina, Beao, JakobVoss, and AnonMoos Open Access logo, converted into svg, designed by PLoS. This version with transparent background. http://commons.wikimedia.org/wiki/File:Open_Access_logo_PLoS_white.svg art designer at PLoS, modified by Wikipedia users Nina, Beao, JakobVoss, and AnonMoos http://www.plos.org/ http://dspace.librar...arrow_drop_down
image/svg+xml art designer at PLoS, modified by Wikipedia users Nina, Beao, JakobVoss, and AnonMoos Open Access logo, converted into svg, designed by PLoS. This version with transparent background. http://commons.wikimedia.org/wiki/File:Open_Access_logo_PLoS_white.svg art designer at PLoS, modified by Wikipedia users Nina, Beao, JakobVoss, and AnonMoos http://www.plos.org/
image/svg+xml art designer at PLoS, modified by Wikipedia users Nina, Beao, JakobVoss, and AnonMoos Open Access logo, converted into svg, designed by PLoS. This version with transparent background. http://commons.wikimedia.org/wiki/File:Open_Access_logo_PLoS_white.svg art designer at PLoS, modified by Wikipedia users Nina, Beao, JakobVoss, and AnonMoos http://www.plos.org/
https://doi.org/10.33540/68...
Doctoral thesis . 2021 . Peer-reviewed
Data sources: Crossref
versions View all 2 versions
addClaim

Personalizing CFTR modulator therapies

Authors: Berkers, Gitte;

Personalizing CFTR modulator therapies

Abstract

over 2000 genetic mutations that can cause CF, resulting in many phenotypes. Recently new drugs were developed that treat the disease at the origin of the problem; they enhance the function of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein which is not functioning properly in CF. These potentially life saving drugs are currently only available for part of the CF population and especially patients with a rare mutation often don’t qualify for a treatment. By culturing stem cells of a patient in the lab, we can generate organoid which can be seen as a sort of mini-organs. This culture technique generates many options for research and individualised drug testing without the need to test a treatment in a patient. Research in this thesis shows how intestinal organoids can be used to treat patients with CF. It shows for example how organoids can be used to identify effective treatments for (groups of) patients. Because of this research a number of CF patients with a rare CFTR mutation was already successfully selected for a life changing treatment. This thesis also provides the basis for a current European study in which treatments are selected for about 2000 European CF patients with a rare CFTR mutation. It is expected that in the near future the organoid model can not only be used for research in patients with CF but also for patients with other diseases.

Country
Netherlands
Related Organizations
Keywords

CFTR; personalized, organoids, modulator, treatment, individual, CF

  • BIP!
    Impact byBIP!
    selected citations
    These citations are derived from selected sources.
    This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
    0
    popularity
    This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
    Average
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
    Average
    impulse
    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
    Average
Powered by OpenAIRE graph
Found an issue? Give us feedback
selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
0
Average
Average
Average
Green
bronze