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Method for screening substance capable of inhibiting abnormal splicing causative of onset or progress of disease
专利权人:
KYOTO UNIVERSITY
发明人:
Masatoshi Hagiwara,Suguru Yoshida,Takamitsu Hosoya
申请号:
US14904609
公开号:
US09879014B2
申请日:
2014.07.14
申请国别(地区):
US
年份:
2018
代理人:
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;
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