发明名称 |
Treatment of cystic fibrosis |
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. |
申请公布号 |
US8410081(B2) |
申请公布日期 |
2013.04.02 |
申请号 |
US20070298506 |
申请日期 |
2007.04.24 |
申请人 |
AERTS JOHANNES MARIA FRANCISCUS GERARDUS;BOOT ROLF GABRIEL;ACADEMISCH MEDISCH CENTRUM |
发明人 |
AERTS JOHANNES MARIA FRANCISCUS GERARDUS;BOOT ROLF GABRIEL |
分类号 |
A01N45/00;A61K31/56 |
主分类号 |
A01N45/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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