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METHOD FOR TREATING MUSCULAR DYSTROPHY BY TARGETING UTROPHIN GENE
METHOD FOR TREATING MUSCULAR DYSTROPHY BY TARGETING UTROPHIN GENE
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机译:靶向麦冬蛋白治疗肌营养不良的方法
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摘要
Polynucleotides comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcription activator, and (b) a base sequence encoding a guide RNA targeting a continuous region of 18 to 24 nucleotides in length in a region set forth in SEQ ID NO: 104, 105, 135, 141, 153, 167, or 172 in the expression regulatory region of human Utrophin gene are expected to be useful for treating or preventing DUCHENNE muscular dystrophy or BECKER muscular dystrophy.
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机译:包含以下碱基序列的多核苷酸:(a)编码核酸酶缺陷型CRISPR效应子蛋白和转录激活因子的融合蛋白的碱基序列,和(b)编码靶向18至24个核苷酸的连续区域的指导RNA的碱基序列预期在人Utrophin基因的表达调节区域中SEQ ID NO:104、105、135、141、153、167或172所示区域中的长度的增加可用于治疗或预防DUCHENNE肌营养不良或BECKER肌营养不良。
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