Treatment of cystic fibrosis
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United States of America Patent
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Apr 2, 2013
Grant Date -
Jul 23, 2009
app pub date -
Apr 24, 2007
filing date -
Apr 24, 2006
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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.
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