Polyamide compound and pharmaceutical composition for treating mitochondrial genetic disease
    3.
    发明授权
    Polyamide compound and pharmaceutical composition for treating mitochondrial genetic disease 有权
    聚酰胺化合物和用于治疗线粒体遗传病的药物组合物

    公开(公告)号:US09518152B2

    公开(公告)日:2016-12-13

    申请号:US14008982

    申请日:2012-04-02

    申请人: Takamitsu Yano

    发明人: Takamitsu Yano

    摘要: 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).

    摘要翻译: 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。

    BIODEGRADABLE POLY (BETA-AMINO ESTERS) AND USES THEREOF
    5.
    发明申请
    BIODEGRADABLE POLY (BETA-AMINO ESTERS) AND USES THEREOF 有权
    生物可降解聚(氨基甲酸酯)及其用途

    公开(公告)号:US20160022821A1

    公开(公告)日:2016-01-28

    申请号:US14811613

    申请日:2015-07-28

    摘要: Poly(β-amino esters) prepared from the conjugate addition of bis(secondary amines) or primary amines to a bis(acrylate ester) are described. Methods of preparing these polymers from commercially available starting materials are also provided. These tertiary amine-containing polymers are preferably biodegradable and biocompatible and may be used in a variety of drug delivery systems. Given the poly(amine) nature of these polymers, they are particularly suited for the delivery of polynucleotides. Nanoparticles containing polymer/polynucleotide complexes have been prepared. The inventive polymers may also be used to encapsulate other agents to be delivered. They are particularly useful in delivering labile agents given their ability to buffer the pH of their surroundings.

    摘要翻译: 描述了从双(仲胺)或伯胺向双(丙烯酸酯)共轭加成制备的聚(&(bgr。 - 氨基酯))。 还提供了从市售的原料制备这些聚合物的方法。 这些含叔胺的聚合物优选是可生物降解的和生物相容的并且可以用于各种药物递送系统中。 鉴于这些聚合物的聚(胺)性质,它们特别适用于递送多核苷酸。 已经制备了含有聚合物/多核苷酸复合物的纳米颗粒。 本发明的聚合物也可以用于包封待递送的其它试剂。 考虑到缓冲其周围环境的pH值,它们特别适用于提供不稳定剂。

    POLYAMIDE COMPOUND AND PHARMACEUTICAL COMPOSITION FOR TREATING MITOCHONDRIAL GENETIC DISEASE
    8.
    发明申请
    POLYAMIDE COMPOUND AND PHARMACEUTICAL COMPOSITION FOR TREATING MITOCHONDRIAL GENETIC DISEASE 有权
    聚酰胺化合物和药物组合物治疗麻醉遗传病

    公开(公告)号:US20150191571A1

    公开(公告)日:2015-07-09

    申请号:US14008982

    申请日:2012-04-02

    申请人: Takamitsu Yano

    发明人: Takamitsu Yano

    IPC分类号: C08G73/06 A61K31/787

    摘要: 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).

    摘要翻译: 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。