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artificial genome manipulation for regulation of gene expression

机译:人工基因组操纵调节基因表达

摘要

the present invention relates to an expression control composition for regulating the expression of repeated genes or a method that uses it. more specifically, the present invention relates to an expression regulation composition comprising a guide nucleic acid with the ability to target the transcriptional regulatory region of repeated genes and a method for regulating the expression of repeated genes using the composition for manipulate and / or artificially modify a transcriptional regulatory region of the repeated genes. in addition, the present invention relates to a method for treating or alleviating a disease caused by gene repetition with the use of an expression control composition to regulate the expression of repeated genes.
机译:本发明涉及用于调节重复基因表达的表达控制组合物或使用它的方法。更具体地,本发明涉及一种表达调控组合物,其包括能够靶向重复基因的转录调控区的指导核酸,以及使用该组合物来操纵和/或人工修饰A,B,C,C,C,C和C的组合物来调控重复基因的表达的方法。重复基因的转录调控区域。另外,本发明涉及通过使用表达控制组合物调节重复基因的表达来治疗或减轻由基因重复引起的疾病的方法。

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