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Methods for inhibiting CAG elongation gene product expression and gene product expression and aggregation, as well as methods for identifying agents that are useful for inhibition
Methods for inhibiting CAG elongation gene product expression and gene product expression and aggregation, as well as methods for identifying agents that are useful for inhibition
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机译:抑制CAG延伸基因产物表达以及基因产物表达和聚集的方法,以及鉴定对抑制有用的药物的方法
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摘要
This invention provides a method for modulating the expression of a first gene in a cell wherein the first gene is one containing more than 36 CAG trinucleotide repeats and encoding a protein that form polyglutamine-mediated protein aggregation. Suppression of the first gene is achieved by reducing the expression of SPT4 gene or SUPT4H gene. It can also be achieved by inhibiting the formation of a Spt4/Spt5 complex or a Supt4h/Supt5h complex. Also provided is a method for identifying an agent useful for modulating the expression and aggregation of CAG-expanded gene product, or treating a polyglutamine disease such as Huntington's disease.
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