Treatment of cystic fibrosis
AssigneeAcademisch Medisch Centrum
InventorsJohannes Maria Franciscus Gerardus Aerts, Rolf Gabriel Boot
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Abstract
The present invention discloses a therapeutic target for the treatment of cystic 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 center (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.
Claims11
A method for treating a subject with cystic fibrosis, comprising administering to a subject in need thereof an effective amount of an inhibitor of glucosidase, beta (bile acid) 2 (GBA2) that comprises:
- (a) a deoxynojirimycin moiety, or a derivative thereof, coupled to
- (b) a 6-40 carbon atom group that inserts into a membrane bilayer, wherein the carbon atom group comprises a hydrophobic moiety which comprises three or more organic ring structures wherein a ring shares two or more carbon atoms with another ring.
Dependencies are inferred from the claim text.