Improved treatment of cystic fibrosis

Details Improved treatment of cystic fibrosis Improved treatment of cystic fibrosis

C - Chemistry, Metallurgy

07

H

C07H 15/12 (2006.01) A61K 31/70 (2006.01) A61P 43/00 (2006.01)

Patent

CA 2650311

The present invention discloses a therapeutic target for the treatment of cyst ic fibrosis. It was found that inhibition of non-lysosomal glucosylceramidase (GBA2) sufficiently restores chloride current in cells from CF patients carrying the common delF508-CFTR mutation. With the catalytic centre (4) of the enzyme positioned on top of the membrane bilayer face particularly potent inhibitors are found in deoxynojirimycin derivatives having a group that is capable of inserting in the membrane bilayer.

L'invention concerne une cible thérapeutique pour le traitement de la fibrose kystique. L'inhibition de la glucosylcéramidase non lysosomale (GBA2) rétablit suffisamment le courant de chlorure dans des cellules de patients souffrant de fibrose kystique (CF) portant la mutation commune delF508-CFTR. Avec le centre catalytique (4) de l'enzyme placée au-dessus de la face de la membrane bicouche, des inhibiteurs particulièrement puissants sont trouvés dans des dérivés désoxynojirimycine ayant un groupe capable de s'introduire dans la membrane bicouche.

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