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GENOME EDITING OF GENES ASSOCIATED WITH TRINUCLEOTIDE REPEAT EXPANSION DISORDERS IN ANIMALS

机译:与动物中三核苷酸重复扩展疾病相关的基因的基因编辑

摘要

The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with trinucleotide repeat expansion disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.
机译:本发明提供了遗传修饰的动物和细胞,其包含编辑的染色体序列,所述染色体序列编码与三核苷酸重复扩增疾病相关的蛋白质。特别地,使用锌指核酸酶介导的编辑过程产生动物或细胞。还提供了使用本文公开的遗传修饰的动物或细胞来筛选试剂的毒性和其他作用的方法。

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