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    Selective reduction of the deleterious activity of extended tri-nucleotide repeat containing genes
    3.
    发明授权
    Selective reduction of the deleterious activity of extended tri-nucleotide repeat containing genes 有权
    延长三核苷酸重复基因的有害活性的选择性降低

    公开(公告)号：US09211303B2

    公开(公告)日：2015-12-15

    申请号：US13988605

    申请日：2011-12-08

    申请人： Tzu-Hao Cheng ,  Chia-Rung Liu ,  Tzu-Han Wang ,  Stanley N. Cohen

    发明人： Tzu-Hao Cheng ,  Chia-Rung Liu ,  Tzu-Han Wang ,  Stanley N. Cohen

    IPC分类号： A61K31/70 ,  C07H21/02 ,  C07H21/04 ,  A61K31/713 ,  C12N15/113 ,  A61K51/10

    CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K51/10 ,  A61K51/1075 ,  C12N2310/14 ,  C12N2320/30 ,  C12Q1/6876 ,  C12Q2600/136 ,  C12Q2600/158 ,  G01N33/5023

    摘要： Aspects of the invention include methods of selectively reducing the deleterious activity of mutant extended trinucleotide repeat containing genes in a cell, as well as compositions used in such methods. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended trinucleotide repeat containing gene may be selectively reduced in a variety of different ways, e.g., by selectively decreasing SPT4 mediated transcriptional activity, by enhancing functionality of proteins encoded thereby, etc. Aspects of the invention further include assays for identifying agents that find use in methods of the invention, e.g. as summarized above. Methods and compositions of the invention find use in a variety of different applications, including the prevention or treatment of disease conditions associated with the presence of genes containing mutant extended trinucleotide repeats, such as Huntington's Disease (HD).

    摘要翻译： 本发明的方面包括选择性地降低细胞中突变型延伸三核苷酸重复含基因的有害活性的方法，以及用于这些方法的组合物。 突变型延伸三核苷酸重复含基因的有害活性（例如，由此编码的产物的毒性和/或功能异常）可以多种不同方式选择性地降低，例如通过增强功能性选择性降低SPT4介导的转录活性 由此编码的蛋白质等。本发明的方面还包括用于鉴定在本发明方法中使用的试剂的测定法，例如 如上所述。 本发明的方法和组合物可用于各种不同的应用，包括预防或治疗与含有突变型延伸三核苷酸重复序列（例如亨廷顿氏病（HD））的基因的存在相关的疾病状况。

    Selective Reduction of the Deleterious Activity of Extended Tri-Nucleotide Repeat Containing Genes
    4.
    发明申请
    Selective Reduction of the Deleterious Activity of Extended Tri-Nucleotide Repeat Containing Genes 有权
    选择性降低扩增的三核苷酸重复包含基因的有害活性

    公开(公告)号：US20130317088A1

    公开(公告)日：2013-11-28

    申请号：US13988605

    申请日：2011-12-08

    申请人： Tzu-Hao Cheng ,  Chia-Rung Liu ,  Tzu-Han Wang ,  Stanley N. Cohen

    发明人： Tzu-Hao Cheng ,  Chia-Rung Liu ,  Tzu-Han Wang ,  Stanley N. Cohen

    IPC分类号： A61K31/713

    CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K51/10 ,  A61K51/1075 ,  C12N2310/14 ,  C12N2320/30 ,  C12Q1/6876 ,  C12Q2600/136 ,  C12Q2600/158 ,  G01N33/5023

    摘要： Aspects of the invention include methods of selectively reducing the deleterious activity of mutant extended trinucleotide repeat containing genes in a cell, as well as compositions used in such methods. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended trinucleotide repeat containing gene may be selectively reduced in a variety of different ways, e.g., by selectively decreasing SPT4 mediated transcriptional activity, by enhancing functionality of proteins encoded thereby, etc. Aspects of the invention further include assays for identifying agents that find use in methods of the invention, e.g. as summarized above. Methods and compositions of the invention find use in a variety of different applications, including the prevention or treatment of disease conditions associated with the presence of genes containing mutant extended trinucleotide repeats, such as Huntington's Disease (HD).

    摘要翻译： 本发明的方面包括选择性地降低细胞中突变型延伸三核苷酸重复含基因的有害活性的方法，以及用于这些方法的组合物。 突变型延伸三核苷酸重复含基因的有害活性（例如，由此编码的产物的毒性和/或功能异常）可以多种不同方式选择性地降低，例如通过增强功能性选择性降低SPT4介导的转录活性 由此编码的蛋白质等。本发明的方面还包括用于鉴定在本发明方法中使用的试剂的测定法，例如 如上所述。 本发明的方法和组合物可用于各种不同的应用，包括预防或治疗与含有突变型延伸三核苷酸重复序列（例如亨廷顿氏病（HD））的基因的存在相关的疾病状况。

    RECOMBINANT PROTEIN AND METHOD OF SCREENING FOR AGENTS THAT MODULATE POLYPEPTIDE AGGREGATION
    5.
    发明申请
    RECOMBINANT PROTEIN AND METHOD OF SCREENING FOR AGENTS THAT MODULATE POLYPEPTIDE AGGREGATION 有权
    重组蛋白和调节多肽聚集的试剂筛选方法

    公开(公告)号：US20080070246A1

    公开(公告)日：2008-03-20

    申请号：US11523090

    申请日：2006-09-19

    申请人： Tzu-Hao Cheng ,  Chia-Fung Liu

    发明人： Tzu-Hao Cheng ,  Chia-Fung Liu

    IPC分类号： C12Q1/68 ,  G01N33/53 ,  C07H21/04 ,  C12P21/06 ,  C12N9/88 ,  C12N5/06

    CPC分类号： G01N33/6896 ,  C07K14/00 ,  C07K2319/43 ,  G01N2500/00 ,  Y10S435/975

    摘要： This invention provides recombinant protein comprising formula as follows: A-B wherein A is polypeptide containing glutamine-rich domain comprising 8-200 glutamine residues and B is color-alteration enzyme modulated by the domain. This invention also provides an expressing vector and a cell produced therefrom. This invention further provides a method and a kit for screening a therapeutic agent for a neurodegenerative disease.

    摘要翻译： 本发明提供了包含如下公式的重组蛋白：<？in-line-formula description =“In-line Formulas”end =“lead”？> AB <？in-line-formula description =“In-line formula”end = “尾”→其中A是含有谷氨酰胺富含结构域的多肽，其含有8-200个谷氨酰胺残基，B是由该域调节的变色酶。 本发明还提供了表达载体和由其制备的细胞。 本发明还提供了用于筛选神经变性疾病的治疗剂的方法和试剂盒。

    Recombinant protein and method of screening for agents that modulate polypeptide aggregation
    6.
    发明授权
    Recombinant protein and method of screening for agents that modulate polypeptide aggregation 有权
    重组蛋白和筛选调节多肽聚集的试剂的方法

    公开(公告)号：US07375190B2

    公开(公告)日：2008-05-20

    申请号：US11523090

    申请日：2006-09-19

    申请人： Tzu-Hao Cheng ,  Chia-Rung Liu

    发明人： Tzu-Hao Cheng ,  Chia-Rung Liu

    IPC分类号： C07K14/00 ,  C12N1/20 ,  C12N15/00 ,  C12Q1/00

    CPC分类号： G01N33/6896 ,  C07K14/00 ,  C07K2319/43 ,  G01N2500/00 ,  Y10S435/975

    摘要： This invention provides recombinant protein comprising formula as follows: A-B wherein A is polypeptide containing glutamine-rich domain comprising 8-200 glutamine residues and B is color-alteration enzyme modulated by the domain. This invention also provides an expressing vector and a cell produced therefrom. This invention further provides a method and a kit for screening a therapeutic agent for a neurodegenerative disease.

    摘要翻译： 本发明提供了包含如下公式的重组蛋白：<？in-line-formula description =“In-line Formulas”end =“lead”？> AB <？in-line-formula description =“In-line formula”end = “尾”→其中A是含有谷氨酰胺富含结构域的多肽，其含有8-200个谷氨酰胺残基，B是由该域调节的变色酶。 本发明还提供了表达载体和由其制备的细胞。 本发明还提供了用于筛选神经变性疾病的治疗剂的方法和试剂盒。