The potential for gene repair via triple helix formation.
The potential for gene repair via triple helix formation.
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DOI：
10.1172/jci19552
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发表时间：
2003-08
期刊：
影响因子：
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通讯作者：
M. Seidman;P. Glazer
中科院分区：
文献类型：
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作者：
M. Seidman;P. Glazer
三聚体形成的寡核苷酸（TFOs）可以以序列特异性的方式结合DNA中的多嘌呤/多嘧啶区域。这种结合的特异性提高了使用三联体结构进行定向基因组修饰的可能性，其最终目标是修复人类细胞中的遗传缺陷。几项研究表明，用tfo处理哺乳动物细胞可以激发DNA修复和重组，从而可以引入所需的序列变化。本文将总结这一领域的最新进展，同时强调在将三联体技术应用于治疗性基因修复之前仍需克服的主要障碍。
Triplex-forming oligonucleotides (TFOs) can bind to polypurine/polypyrimidine regions in DNA in a sequence-specific manner. The specificity of this binding raises the possibility of using triplex formation for directed genome modification, with the ultimate goal of repairing genetic defects in human cells. Several studies have demonstrated that treatment of mammalian cells with TFOs can provoke DNA repair and recombination, in a manner that can be exploited to introduce desired sequence changes. This review will summarize recent advances in this field while also highlighting major obstacles that remain to be overcome before the application of triplex technology to therapeutic gene repair can be achieved.