发明名称 |
NOVEL CYSTIC FIBROSIS TREATMENT |
摘要 |
The present invention provides a method of stimulating expression of a functional full-length cystic fibrosis transmembrane conductance regulator protein in an individual in need of such treatment, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside. Also provided is a method of treating cystic fibrosis in an individual in need of such treatment, comprising the step of administering to said individual a therapeutically effective dose of an aminoglycoside. Further provided is a method of screening for a drug useful in the treatment of an individual having cystic fibrosis, comprising the step of determining said drug's ability to suppress premature stop mutations in a model of cystic fibrosis and a method of pharmacologically suppressing premature stop mutations in an individual having such mutations, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside.
|
申请公布号 |
WO9734611(A1) |
申请公布日期 |
1997.09.25 |
申请号 |
WO1997US03464 |
申请日期 |
1997.03.21 |
申请人 |
UAB RESEARCH FOUNDATION;BEDWELL, DAVID, M. |
发明人 |
BEDWELL, DAVID, M. |
分类号 |
A61K31/70;(IPC1-7):A61K31/70 |
主分类号 |
A61K31/70 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|