Compounds for the Modulation of Huntingtin Aggregation, Methods and Means for Identifying Such Compounds
    2.
    发明申请
    Compounds for the Modulation of Huntingtin Aggregation, Methods and Means for Identifying Such Compounds 审中-公开
    用于调节亨廷顿蛋白聚集的化合物,用于鉴定这些化合物的方法和方法

    公开(公告)号:US20100298280A1

    公开(公告)日:2010-11-25

    申请号:US12602200

    申请日:2008-06-13

    摘要: The present invention relates to tetranortriterpenoid compounds and pharmaceutical compositions thereof, which are provided for use in the treatment, diagnosis and/or prevention of trinucleotide repeat disorders (like a polyglutamine diseases, e.g Huntingdon's disease), amyloid diseases, neurodegenerative disease, protein misfolding diseases or tumors. The tetranortriterpenoid compounds of the present invention are further provided for the reduction and/or inhibition of the aggregation of amyloidogenic proteins, preferably of polyglutamine proteins (such as huntingtin) as well as for increasing proteasome activity. The present invention furthermore relates to nucleic acids, comprising the nucleotide sequences of two huntingtin fragments, as well as to cells and kits, which are useful in methods for assessing the aggregation of huntingtin and in methods for identifying compounds, which modulate the aggregation of huntingtin.

    摘要翻译: 本发明涉及四唑类萜类化合物及其药物组合物,其用于治疗,诊断和/或预防三核苷酸重复病症(如聚谷氨酸病,如亨廷顿氏病),淀粉样蛋白病,神经变性疾病,蛋白质错误折叠疾病 或肿瘤。 进一步提供本发明的四硝酸萜类化合物用于还原和/或抑制淀粉样蛋白生成蛋白,优选聚谷氨酸蛋白(例如亨廷顿蛋白)的聚集以及用于增加蛋白酶体活性。 本发明还涉及包含两个亨廷顿蛋白片段的核苷酸序列的核酸,以及可用于评价亨廷汀蛋白聚集的方法的细胞和试剂盒,以及用于鉴定调节亨廷顿蛋白聚集的化合物的方法 。