Prophylactic or Ameliorating Agent for Genetic Diseases
    1.
    发明申请
    Prophylactic or Ameliorating Agent for Genetic Diseases 有权
    遗传疾病预防或改善剂

    公开(公告)号:US20130102644A1

    公开(公告)日:2013-04-25

    申请号:US13805151

    申请日:2011-06-27

    IPC分类号: A61K31/428

    摘要: An object of the present invention is to provide a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation. The prophylactic or ameliorating agent used in the present invention is a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation, wherein the prophylactic or ameliorating agent contains a compound having a molecular weight of 1500 or lower.

    摘要翻译: 本发明的目的是提供由基因外显子突变引起的遗传疾病的预防或改善剂,并且能够通过跳过包含突变的外显子形成功能性截短的蛋白质。 用于本发明的预防或改善剂是由基因外显子突变引起的遗传疾病的预防或改善剂,能够通过跳过包含突变的外显子形成功能性截短蛋白, 其中所述预防或改善剂含有分子量为1500以下的化合物。

    Prophylactic or ameliorating agent for genetic diseases
    5.
    发明授权
    Prophylactic or ameliorating agent for genetic diseases 有权
    遗传疾病预防或改善剂

    公开(公告)号:US09241929B2

    公开(公告)日:2016-01-26

    申请号:US13805151

    申请日:2011-06-27

    摘要: An object of the present invention is to provide a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation. The prophylactic or ameliorating agent used in the present invention is a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation, wherein the prophylactic or ameliorating agent contains a compound having a molecular weight of 1500 or lower.

    摘要翻译: 本发明的目的是提供由基因外显子突变引起的遗传疾病的预防或改善剂,并且能够通过跳过包含突变的外显子形成功能性截短的蛋白质。 用于本发明的预防或改善剂是由基因外显子突变引起的遗传疾病的预防或改善剂,能够通过跳过包含突变的外显子形成功能性截短蛋白, 其中所述预防或改善剂含有分子量为1500以下的化合物。