发明名称 |
METHOD OF TREATING GENETIC DISEASE CAUSED BY NONSENSE MUTATION |
摘要 |
<p>It is intended to provide a compound having a readthrough activity to be used as a method of treating a genetic disease caused by a nonsense mutation, a pharmaceutical composition containing the compound, and a method of treating a genetic disease caused by a nonsense mutation including administering the compound. According to the invention, the method which is a method in which a wild-type normal protein is produced in the body of a mammal from a gene in which a premature stop codon is generated by a mutation and includes administering a compound represented by the following formula (VI): (VI) (wherein R<SUP>1</SUP>, R<SUP>2</SUP>, R<SUP>3</SUP>, R<SUP>4</SUP>, R<SUP>5</SUP> and X<SUP>1</SUP> are as defined in the description) or the like to the mammal is provided.</p> |
申请公布号 |
WO2008004610(A1) |
申请公布日期 |
2008.01.10 |
申请号 |
WO2007JP63436 |
申请日期 |
2007.07.05 |
申请人 |
THE UNIVERSITY OF TOKYO;UNIVERSITY OF MARYLAND, BALTIMORE;MATSUDA, RYOICHI;SHIOZUKA, MASATAKA;MACKERELL, JR., ALEXANDER |
发明人 |
MATSUDA, RYOICHI;SHIOZUKA, MASATAKA;MACKERELL, JR., ALEXANDER |
分类号 |
A61P21/04;A61K31/195;A61K31/197;A61K31/221;A61K38/00;A61P43/00;C12N15/09 |
主分类号 |
A61P21/04 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|