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Assessment of p.Phe508del-CFTR functional restoration in pediatric primary cystic fibrosis airway epithelial cells.

Author
Abstract
:

Mutations in the cystic fibrosis transmembrane regulator (CFTR) gene can reduce function of the CFTR ion channel activity and impair cellular chloride secretion. The gold standard method to assess CFTR function of ion transport using the Ussing chamber requires a high number of airway epithelial cells grown at air-liquid interface, limiting the application of this method for high throughput screening of potential therapeutic compounds in primary airway epithelial cells (pAECs) featuring less common CFTR mutations. This study assessed an alternative approach, using a small scale halide assay that can be adapted for a personalized high throughput setting to analyze CFTR function of pAEC.

Year of Publication
:
0
Journal
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PloS one
Volume
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13
Issue
:
1
Number of Pages
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e0191618
Date Published
:
2018
URL
:
http://dx.plos.org/10.1371/journal.pone.0191618
DOI
:
10.1371/journal.pone.0191618
Short Title
:
PLoS One
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