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    • 4. 发明授权
    • Method for reducing protein misfolding and accumulation in Huntington'S disease cells
    • 减少亨廷顿病细胞中蛋白质错误折叠和积聚的方法
    • US09265786B1
    • 2016-02-23
    • US13854809
    • 2013-04-01
    • University of Wyoming
    • Jonathan H. Fox
    • A61K48/00A01N63/00A61K31/711
    • A61K31/711A61K48/005C12N9/0036C12N9/90C12N15/86C12N2740/16043C12Y503/04001
    • A method and virus for decreasing the levels of the disease-causing protein in Huntington's disease (mhtt) are described. Increased expression of human TXNDC10, and TXN1 in diseased cells decreases levels of toxic mhtt in cell models of disease. Genetically increasing the expression of the genes producing these proteins using viral vectors, or small molecule chemical activators of gene expression or stimulators of enzymatic activity, may be used to treat Huntington's disease in the asymptomatic carrier or affected human. Since other protein misfolding neurodegenerative diseases have many features in common with Huntington's disease, including the structure of the misfolded protein, the present method may be applicable to other protein misfolding neurodegenerative diseases.
    • 描述了用于降低亨廷顿氏病(mhtt)中引起疾病​​的蛋白质的水平的方法和病毒。 人类TXNDC10和TXN1在患病细胞中的表达增加可以降低疾病细胞模型中毒性mhtt的水平。 使用病毒载体或基因表达的小分子化学活化剂或酶活性刺激剂基因上增加产生这些蛋白质的基因的表达,可用于在无症状的载体或受影响的人中治疗亨廷顿舞蹈病。 由于其他蛋白质错误折叠的神经变性疾病具有许多与亨廷顿疾病相关的特征,包括错误折叠的蛋白质的结构,本方法可适用于其他蛋白质错误折叠的神经变性疾病。