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首页> 外文期刊>Neuron >Targeted gene repair and its application to neurodegenerative disorders.
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Targeted gene repair and its application to neurodegenerative disorders.

机译:靶向基因修复及其在神经退行性疾病中的应用。

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摘要

Synthetic DNA oligonucleotides can direct the exchange of single nucleotides within coding regions of mammalian genes by hybridizing to their complementary sequence in the chromosome and creating a recombination joint structure with a single mismatched base pair. Inherent DNA repair processes recognize the mismatch and resolve it using the DNA sequence of the oligonucleotide vector as the template. This gene surgery approach can be used to repair mutations or to disrupt tri-nucleotide repeats in dysfunctional genes responsible for neurological disorders.
机译:合成的DNA寡核苷酸可通过与染色体中的互补序列杂交并创建具有单个错配碱基对的重组关节结构,来指导哺乳动物基因编码区内的单个核苷酸交换。固有的DNA修复过程可以识别错配并使用寡核苷酸载体的DNA序列作为模板来解决。这种基因手术方法可用于修复导致神经系统疾病的功能失调的基因中的突变或破坏三核苷酸重复。

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