Johannes Maria Franciscus Gerardus Aerts;Rolf Gabriel Boot
发明人:
Johannes Maria Franciscus Gerardus Aerts,Rolf Gabriel Boot
申请号：
US12298506
公开号：
US08410081B2
申请日:
2007.04.24
申请国别(地区):
US
年份:
2013
代理人:
摘要：
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.
