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ALLELE-SPECIFIC THERAPY FOR HUNTINGTON DISEASE HAPLOTYPES
ALLELE-SPECIFIC THERAPY FOR HUNTINGTON DISEASE HAPLOTYPES
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机译:亨廷顿疾病的等位基因特异性疗法单倍型
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摘要
The present invention relates to compositions and methods of use thereof for inhibiting mutant HTT mRNA transcription or CAG-expanded HTT protein expression in a cell, comprising contacting the cell with an effective amount of an oligomer targeting a differentiating polymorphism, wherein the differentiating polymorphism is selected from rs72239206, rs363107, rs362313, rs2530595, rs113407847. Specific oligomer sequences are also provided.
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