READ THROUGH INDUCER, AND THERAPEUTIC AGENT FOR NONSENSE-MUTATION-TYPE GENETIC DISEASES

Title:

READ THROUGH INDUCER, AND THERAPEUTIC AGENT FOR NONSENSE-MUTATION-TYPE GENETIC DISEASES

Document Type and Number:

WIPO Patent Application WO/2011/096484

Kind Code:

A1

Abstract:

A read through inducer for an immature termination codon formed by a nonsense mutation, which comprises a compound represented by structure formula (A); and a therapeutic agent for nonsense-mutation-type genetic diseases, which comprises the read through inducer.

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Inventors:

MATSUDA RYOICHI (JP)
SHIOZUKA MASATAKA (JP)
WAGATSUMA AKIRA (JP)
TAKAHASHI YOSHIKAZU (JP)
IKEDA DAISHIRO (JP)
NONOMURA YOSHIAKI (JP)
MATSUO MASAFUMI (JP)
NISHIDA ATSUSHI (JP)

Application Number:

PCT/JP2011/052263

Publication Date:

August 11, 2011

Filing Date:

February 03, 2011

Export Citation:

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Assignee:

MICROBIAL CHEM RES FOUND (JP)
UNIV TOKYO (JP)
MATSUDA RYOICHI (JP)
SHIOZUKA MASATAKA (JP)
WAGATSUMA AKIRA (JP)
TAKAHASHI YOSHIKAZU (JP)
IKEDA DAISHIRO (JP)
NONOMURA YOSHIAKI (JP)
MATSUO MASAFUMI (JP)
NISHIDA ATSUSHI (JP)

International Classes:

A61K31/7036; A61P1/16; A61P3/04; A61P3/06; A61P3/10; A61P5/14; A61P7/04; A61P9/02; A61P9/10; A61P11/00; A61P13/12; A61P17/00; A61P19/00; A61P19/02; A61P21/04; A61P25/00; A61P25/16; A61P25/28; A61P27/02; A61P29/00; A61P35/00; A61P37/02; A61P37/06; A61P43/00; C07H15/234

Domestic Patent References:

WO2009019504A1	2009-02-12
WO2008023462A1	2008-02-28
WO2008004610A1	2008-01-10
WO2002102361A1	2002-12-27
WO2002102361A1	2002-12-27
WO2008004610A1	2008-01-10

Foreign References:

JP2009523775A	2009-06-25
JP2009532461A	2009-09-10
JP2004522763A	2004-07-29
JP2009255077A	2009-11-05
JP2009095436A	2009-05-07
JP2008161075A	2008-07-17
JP2008079510A	2008-04-10

Other References:

MARINA MANUVAKHOVA ET AL.: "Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system", RNA, vol. 6, 2000, pages 1044 - 1055, XP008167504
MASAYUKI ARAKAWA ET AL.: "Kin- Dystrophy ni Taisuru Yakubutsu Chiryo", IGAKU NO AYUMI, vol. 204, no. 3, 8 January 2003 (2003-01-08), pages 183 - 188, XP008167502
ACTA. MYOL., vol. 22, 2003, pages 15 - 21
N. ENGL. J. MED., vol. 349, 2003, pages 1433 - 1441
THE JOURNAL OF ANTIBIOTICS, vol. 26, 1973, pages 412
"Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations.", J. MED. CHEM., vol. 52, 2009, pages 2836 - 2845
FUJISAWA, K.; HOSHIYA, T.; KAWAGUCHI, H.: "Aminoglycoside antibiotics. VII. Acute toxicity of aminoglycoside antibiotics.", J. ANTIBIOT., vol. 27, 1974, pages 677 - 681
KONDO, S.; HOTTA. K.: "Semisvnthetic aminoglvcoside antibiotics:Development anr! enzymatic modifications.", J. INFECT. CHEMOTHER, vol. 5, 1999, pages 1 - 9
See also references of EP 2535051A4

Attorney, Agent or Firm:

HIROTA, Koichi et al. (JP)
Koichi Hirota (JP)

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