Cystic fibrosis research

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat” (FEBS Lett. 2006 Mar 10). Authors are Norez C, Noel S, Wilke M et al., from the Institut de Physiologie et Biologie Cellulaires, CNRS UMR 6187, Universite de Poitiers, France. In the disease cystic fibrosis (CF), the most common mutation delF508 results in endoplasmic reticulum retention of misfolded CF gene proteins (CFTR). The authors 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. Since miglustat is an orally active orphan drug (Zavesca((R))) prescribed for the treatment of Gaucher disease, these findings provide the basis for future clinical evaluation of miglustat in CF patients. To access the full abstract of the article, click here.