dc.contributor.author
Graeber, Simon Y.
dc.contributor.author
Vitzthum, Constanze
dc.contributor.author
Mall, Marcus A.
dc.date.accessioned
2021-11-02T10:35:59Z
dc.date.available
2021-11-02T10:35:59Z
dc.identifier.uri
https://refubium.fu-berlin.de/handle/fub188/32467
dc.identifier.uri
http://dx.doi.org/10.17169/refubium-32192
dc.description.abstract
Refinement of personalized treatment of cystic fibrosis (CF) with emerging medicines targeting the CF basic defect will likely benefit from biomarkers sensitive to detect improvement of cystic fibrosis transmembrane conductance regulator (CFTR) function in individual patients. Intestinal current measurement (ICM) is a technique that enables quantitative assessment of CFTR chloride channel function in rectal tissues or other intestinal epithelia. ICM was originally developed to study the CF ion transport defect in the intestine and has been established as a sensitive biomarker of CFTR function and diagnostic test for CF. With the emergence of CFTR-directed therapeutics, ICM has become an important tool to estimate the level of rescue of CFTR function achieved by approved CFTR modulators, both at the level of CFTR genotype groups, as well as individual patients with CF. In combination with preclinical patient-derived cell culture models, ICM may aid the development of targeted therapies for patients with rare CFTR mutations. Here, we review the principles of ICM and examine how this CFTR biomarker may be used to support diagnostic testing and enhance personalized medicine for individual patients with common as well as rare CFTR mutations in the new era of medicines targeting the underlying cause of CF.
en
dc.rights.uri
https://creativecommons.org/licenses/by/4.0/
dc.subject
cystic fibrosis
en
dc.subject
intestinal current measurement (ICM)
en
dc.subject
personalized medicine
en
dc.subject.ddc
600 Technik, Medizin, angewandte Wissenschaften::610 Medizin und Gesundheit::610 Medizin und Gesundheit
dc.title
Potential of Intestinal Current Measurement for Personalized Treatment of Patients with Cystic Fibrosis
dc.type
Wissenschaftlicher Artikel
dcterms.bibliographicCitation.articlenumber
384
dcterms.bibliographicCitation.doi
10.3390/jpm11050384
dcterms.bibliographicCitation.journaltitle
Journal of Personalized Medicine
dcterms.bibliographicCitation.number
5
dcterms.bibliographicCitation.originalpublishername
MDPI AG
dcterms.bibliographicCitation.volume
11
refubium.affiliation
Charité - Universitätsmedizin Berlin
refubium.resourceType.isindependentpub
no
dcterms.accessRights.openaire
open access
dcterms.bibliographicCitation.pmid
34066648
dcterms.isPartOf.eissn
2075-4426