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&lt;SUP&gt;1&lt;/SUP&gt;, R&lt;SUP&gt;2&lt;/SUP&gt;, R&lt;SUP&gt;3&lt;/SUP&gt;, R&lt;SUP&gt;4&lt;/SUP&gt;, R&lt;SUP&gt;5&lt;/SUP&gt; and X&lt;SUP&gt;1&lt;/SUP&gt; 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