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Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat.
FEBS Lett. 2006 Apr 03; 580(8):2081-6.FL

Abstract

In the disease cystic fibrosis (CF), the most common mutation delF508 results in endoplasmic reticulum retention of misfolded CF gene proteins (CFTR). We show that the alpha-1,2-glucosidase inhibitor miglustat (N-butyldeoxynojirimycin, NB-DNJ) prevents delF508-CFTR/calnexin interaction and restores cAMP-activated chloride current in epithelial CF cells. Moreover, miglustat rescues a mature and functional delF508-CFTR in the intestinal crypts of ileal mucosa from delF508 mice. Since miglustat is an orally active orphan drug (Zavesca) prescribed for the treatment of Gaucher disease, our findings provide the basis for future clinical evaluation of miglustat in CF patients.

Authors+Show Affiliations

Institut de Physiologie et Biologie Cellulaires, CNRS UMR 6187, Université de Poitiers, 40 avenue du recteur Pineau, Poitiers 86022, France. cnorez@ext.univ-poitiers.frNo affiliation info availableNo affiliation info availableNo affiliation info availableNo 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

16546175

Citation

Norez, Caroline, et al. "Rescue of Functional delF508-CFTR Channels in Cystic Fibrosis Epithelial Cells By the Alpha-glucosidase Inhibitor Miglustat." FEBS Letters, vol. 580, no. 8, 2006, pp. 2081-6.
Norez C, Noel S, Wilke M, et al. Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. FEBS Lett. 2006;580(8):2081-6.
Norez, C., Noel, S., Wilke, M., Bijvelds, M., Jorna, H., Melin, P., DeJonge, H., & Becq, F. (2006). Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. FEBS Letters, 580(8), 2081-6.
Norez C, et al. Rescue of Functional delF508-CFTR Channels in Cystic Fibrosis Epithelial Cells By the Alpha-glucosidase Inhibitor Miglustat. FEBS Lett. 2006 Apr 3;580(8):2081-6. PubMed PMID: 16546175.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. AU - Norez,Caroline, AU - Noel,Sabrina, AU - Wilke,Martina, AU - Bijvelds,Marcel, AU - Jorna,Huub, AU - Melin,Patricia, AU - DeJonge,Hugo, AU - Becq,Frederic, Y1 - 2006/03/10/ PY - 2006/02/07/received PY - 2006/03/01/revised PY - 2006/03/06/accepted PY - 2006/3/21/pubmed PY - 2006/5/5/medline PY - 2006/3/21/entrez SP - 2081 EP - 6 JF - FEBS letters JO - FEBS Lett. VL - 580 IS - 8 N2 - In the disease cystic fibrosis (CF), the most common mutation delF508 results in endoplasmic reticulum retention of misfolded CF gene proteins (CFTR). We show that the alpha-1,2-glucosidase inhibitor miglustat (N-butyldeoxynojirimycin, NB-DNJ) prevents delF508-CFTR/calnexin interaction and restores cAMP-activated chloride current in epithelial CF cells. Moreover, miglustat rescues a mature and functional delF508-CFTR in the intestinal crypts of ileal mucosa from delF508 mice. Since miglustat is an orally active orphan drug (Zavesca) prescribed for the treatment of Gaucher disease, our findings provide the basis for future clinical evaluation of miglustat in CF patients. SN - 0014-5793 UR - https://www.unboundmedicine.com/medline/citation/16546175/Rescue_of_functional_delF508_CFTR_channels_in_cystic_fibrosis_epithelial_cells_by_the_alpha_glucosidase_inhibitor_miglustat_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0014-5793(06)00304-8 DB - PRIME DP - Unbound Medicine ER -