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    • 6. 发明授权
    • Polyamide compound and pharmaceutical composition for treating mitochondrial genetic disease
    • 聚酰胺化合物和用于治疗线粒体遗传病的药物组合物
    • US09518152B2
    • 2016-12-13
    • US14008982
    • 2012-04-02
    • Takamitsu Yano
    • Takamitsu Yano
    • C07D403/14C08G73/06A61K31/785A61K31/787C12N15/10A61K47/48
    • A61K31/4178A61K31/785A61K31/787A61K47/595C07D403/14C08G73/0611C08G73/0616C12N15/1062C12Q2600/178
    • The object of the present invention is to provide a fundamental therapy to mitochondrial genetic diseases caused by mutation of the mitochondrial (mt)DNA, and a pharmaceutical composition used for the same. The object can be solved by a polyamide compound binding to a target double-stranded mtDNA comprising A/T pair consisting of first A of the following sense-stranded DNA and the corresponding T, A/T pair consisting of 8th A of the following sense-stranded DNA and the corresponding T, G/C pair consisting of 9th G of the following sense-stranded DNA and the corresponding C, G/C pair consisting of 14th G of the following sense-stranded DNA and the corresponding C, T/A pair consisting of 15th T of the following sense-stranded DNA and the corresponding A, or the like, in the double-stranded DNA consisting of the sense-stranded DNA having base sequence of 5′-ATGGCAGAGCCCGGTAATCGCATAA-3′ (SEQ ID NO: 1) and the antisense-stranded DNA having base sequence of 5′-TTATGCGATTACCGGGCTCTGCCAT-3′ (SEQ ID NO: 2).
    • 本发明的目的是提供对由线粒体(mt)DNA的突变引起的线粒体遗传病的基础疗法和用于其的药物组合物。 该目的可以通过结合到包含由以下正义链DNA的第一个A组成的A / T对的对象双链mtDNA的聚酰胺化合物和由以下意义的第8位组成的对应的T,A / T对 并且相应的T,G / C对由以下正义链DNA的第9G组成的相应的C,G / C对由以下正义链DNA的第14位和对应的C,T / 由具有5'-ATGGCAGAGCCCGGTAATCGCATAA-3'(SEQ ID NO:3)的碱基序列的有义链DNA组成的双链DNA中的由第15位以下的正义链和相应的A等构成的对 :1)和具有5'-TTATGCGATTACCGGGCTCTGCCAT-3'(SEQ ID NO:2)的碱基序列的反义链DNA。
    • 9. 发明申请
    • POLYAMIDE COMPOUND AND PHARMACEUTICAL COMPOSITION FOR TREATING MITOCHONDRIAL GENETIC DISEASE
    • 聚酰胺化合物和药物组合物治疗麻醉遗传病
    • US20150191571A1
    • 2015-07-09
    • US14008982
    • 2012-04-02
    • Takamitsu Yano
    • Takamitsu Yano
    • C08G73/06A61K31/787
    • A61K31/4178A61K31/785A61K31/787A61K47/595C07D403/14C08G73/0611C08G73/0616C12N15/1062C12Q2600/178
    • The object of the present invention is to provide a fundamental therapy to mitochondrial genetic diseases caused by mutation of the mitochondrial (mt)DNA, and a pharmaceutical composition used for the same.The object can be solved by a polyamide compound binding to a target double-stranded mtDNA comprising A/T pair consisting of first A of the following sense-stranded DNA and the corresponding T, A/T pair consisting of 8th A of the following sense-stranded DNA and the corresponding T, G/C pair consisting of 9th G of the following sense-stranded DNA and the corresponding C, G/C pair consisting of 14th G of the following sense-stranded DNA and the corresponding C, T/A pair consisting of 15th T of the following sense-stranded DNA and the corresponding A, or the like, in the double-stranded DNA consisting of the sense-stranded DNA having base sequence of 5′-ATGGCAGAGCCCGGTAATCGCATAA-3′ (SEQ ID NO: 1) and the antisense-stranded DNA having base sequence of 5′-TTATGCGATTACCGGGCTCTGCCAT-3′ (SEQ ID NO: 2).
    • 本发明的目的是提供对由线粒体(mt)DNA的突变引起的线粒体遗传病的基础疗法和用于其的药物组合物。 该目的可以通过结合到包含由以下正义链DNA的第一个A组成的A / T对的对象双链mtDNA的聚酰胺化合物和由以下意义的第8位组成的对应的T,A / T对 并且相应的T,G / C对由以下正义链DNA的第9G组成的相应的C,G / C对由以下正义链DNA的第14位和对应的C,T / 由具有5'-ATGGCAGAGCCCGGTAATCGCATAA-3'(SEQ ID NO:3)的碱基序列的有义链DNA组成的双链DNA中的由第15位以下的正义链和相应的A等构成的对 :1)和具有5'-TTATGCGATTACCGGGCTCTGCCAT-3'(SEQ ID NO:2)的碱基序列的反义链DNA。