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Title:
METHOD FOR SCREENING SUBSTANCE CAPABLE OF INHIBITING ABNORMAL SPLICING CAUSATIVE OF ONSET OR PROGRESS OF DISEASE
Document Type and Number:
WIPO Patent Application WO/2015/005491
Kind Code:
A1
Abstract:
Provided are a compound and a medicinal composition relating to a disease in which an abnormal splice variant participates, utilization of the same, and a method for searching for the same. In one or more embodiments, provided is a compound represented by formula (I) or (I'), a prodrug thereof or a pharmaceutically acceptable salt of the same. In one or more other embodiments, provided is a screening method using a DNA construct that has been fused and arranged or composed so as to express different reporter genes between a wild type splice variant and an abnormal splice variant causative of the onset or progress of a disease.

Inventors:
HAGIWARA MASATOSHI (JP)
YOSHIDA SUGURU (JP)
HOSOYA TAKAMITSU (JP)
Application Number:
PCT/JP2014/068691
Publication Date:
January 15, 2015
Filing Date:
July 14, 2014
Export Citation:
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Assignee:
UNIV KYOTO (JP)
NAT UNIV CORP TOKYO MED & DENT (JP)
International Classes:
C12Q1/68; A61K31/52; A61P25/02; A61P43/00; C07D473/40; C12N15/09; C12N15/11; C12Q1/06
Domestic Patent References:
WO2011152043A12011-12-08
WO2008073619A22008-06-19
WO2010118367A22010-10-14
WO2011152043A12011-12-08
WO2010118367A22010-10-14
Foreign References:
US20060014763A12006-01-19
US6413974B12002-07-02
JP2002504553A2002-02-12
US7737110B22010-06-15
Other References:
J.HOYER ET AL.: "Peptide Vectors for the Nonviral Delivery of Nucleic Acids", ACCOUNTS OF CHEMICAL RESEARCH, vol. 45, no. 7, 2012, pages 1048 - 1056, XP055306549
S.R.BRESHEARS ET AL.: "Purines. VIII. The Aminolysis of Certain Chlorosubstituted Purines", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 81, 1959, pages 3789 - 3792, XP000917842
CHRISOFK ET AL., NATURE (LETTERS, 2008
NOJIMA ET AL., NUCLEIC ACID RESEARCH, 2009
NOJIMA ET AL., NUCLEIC ACIDS RESEARCH, 2009
See also references of EP 3020829A4
Attorney, Agent or Firm:
IKEUCHI SATO & PARTNER PATENT ATTORNEYS (JP)
Patent business corporation Ikeuchi and Sato and partners (JP)
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