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A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype.
Am J Respir Cell Mol Biol. 2009 Aug; 41(2):217-25.AJ

Abstract

Cystic fibrosis (CF) is a fatal, autosomal and recessive genetic disease that is mainly due to inactivating mutations in the chloride channel CF transmembrane conductance regulator (CFTR). Sodium hyperabsorption by the airways, profound lung inflammation, and dysregulation of calcium homeostasis, are presumably causally related to loss of CFTR-dependent chloride function in patients with CF. Miglustat (N-butyldeoxynojirimycin, Zavesca), an inhibitor of the alpha-1,2 glucosidase, has been proposed for clinical use in CF because of its effect as a corrector of the defective trafficking of F508del-CFTR. In the present study, we show that daily treatment for 2 months with low concentrations of miglustat on the human CF nasal epithelial cell line, JME/CF15 (F508del/F508del-CFTR), results in progressive, stable, reversible, and sustained correction of F508del-CFTR trafficking, down-regulation of sodium hyperabsorption, and regulation of the calcium homeostasis. In conclusion, we provide here the first evidence that a respiratory CF cell can acquire a non-CF-like phenotype when chronically treated with low concentrations of a pharmacological drug.

Authors+Show Affiliations

IPBC, Université de Poitiers, 40 Avenue du Recteur Pineau, 86022 Poitiers, France.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

19131642

Citation

Norez, Caroline, et al. "A Cystic Fibrosis Respiratory Epithelial Cell Chronically Treated By Miglustat Acquires a Non-cystic Fibrosis-like Phenotype." American Journal of Respiratory Cell and Molecular Biology, vol. 41, no. 2, 2009, pp. 217-25.
Norez C, Antigny F, Noel S, et al. A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype. Am J Respir Cell Mol Biol. 2009;41(2):217-25.
Norez, C., Antigny, F., Noel, S., Vandebrouck, C., & Becq, F. (2009). A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype. American Journal of Respiratory Cell and Molecular Biology, 41(2), 217-25. https://doi.org/10.1165/rcmb.2008-0285OC
Norez C, et al. A Cystic Fibrosis Respiratory Epithelial Cell Chronically Treated By Miglustat Acquires a Non-cystic Fibrosis-like Phenotype. Am J Respir Cell Mol Biol. 2009;41(2):217-25. PubMed PMID: 19131642.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype. AU - Norez,Caroline, AU - Antigny,Fabrice, AU - Noel,Sabrina, AU - Vandebrouck,Clarisse, AU - Becq,Frédéric, Y1 - 2009/01/08/ PY - 2009/1/10/entrez PY - 2009/1/10/pubmed PY - 2009/8/12/medline SP - 217 EP - 25 JF - American journal of respiratory cell and molecular biology JO - Am. J. Respir. Cell Mol. Biol. VL - 41 IS - 2 N2 - Cystic fibrosis (CF) is a fatal, autosomal and recessive genetic disease that is mainly due to inactivating mutations in the chloride channel CF transmembrane conductance regulator (CFTR). Sodium hyperabsorption by the airways, profound lung inflammation, and dysregulation of calcium homeostasis, are presumably causally related to loss of CFTR-dependent chloride function in patients with CF. Miglustat (N-butyldeoxynojirimycin, Zavesca), an inhibitor of the alpha-1,2 glucosidase, has been proposed for clinical use in CF because of its effect as a corrector of the defective trafficking of F508del-CFTR. In the present study, we show that daily treatment for 2 months with low concentrations of miglustat on the human CF nasal epithelial cell line, JME/CF15 (F508del/F508del-CFTR), results in progressive, stable, reversible, and sustained correction of F508del-CFTR trafficking, down-regulation of sodium hyperabsorption, and regulation of the calcium homeostasis. In conclusion, we provide here the first evidence that a respiratory CF cell can acquire a non-CF-like phenotype when chronically treated with low concentrations of a pharmacological drug. SN - 1535-4989 UR - https://www.unboundmedicine.com/medline/citation/19131642/A_cystic_fibrosis_respiratory_epithelial_cell_chronically_treated_by_miglustat_acquires_a_non_cystic_fibrosis_like_phenotype_ L2 - http://www.atsjournals.org/doi/full/10.1165/rcmb.2008-0285OC?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -