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Method for screening substance capable of inhibiting abnormal splicing causative of onset or progress of disease

机译:能够抑制引起疾病发作或进展的异常剪接的物质的筛选方法

摘要

A compound and a pharmaceutical composition for disease associated with an abnormal splice variant, use of the compound and the pharmaceutical composition, or a screening method of the compound and the pharmaceutical composition are provided. One or more embodiments disclose a compound expressed by the following formula (I) or (I') or prodrugs or pharmaceutically acceptable salts thereof. Another one or more embodiments disclose a screening method using a DNA construct that is fused, arranged, or constructed so as to express different reporter genes for a wild-type splice variant and an abnormal splice variant that contributes to the development or progression of disease;
机译:提供了与异常剪接变体有关的疾病的化合物和药物组合物,该化合物和药物组合物的用途或该化合物和药物组合物的筛选方法。一个或多个实施方式公开了由下式(I)或(I')表示的化合物或其前药或药学上可接受的盐。另一个或多个实施方案公开了使用DNA构建体的筛选方法,所述DNA构建体被融合,布置或构建以便表达有助于野生型剪接变体和异常剪接变体的不同报道基因,所述报道基因有助于疾病的发展或进展。

著录项

  • 公开/公告号IL243564D0

    专利类型

  • 公开/公告日2016-03-31

    原文格式PDF

  • 申请/专利权人 KYOTO UNIVERSITY;

    申请/专利号IL20160243564

  • 发明设计人

    申请日2016-01-11

  • 分类号C12Q;

  • 国家 IL

  • 入库时间 2022-08-21 14:26:00

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