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The potential for gene repair via triple helix formation
Michael M. Seidman, Peter M. Glazer
Michael M. Seidman, Peter M. Glazer
Published August 15, 2003
Citation Information: J Clin Invest. 2003;112(4):487-494. https://doi.org/10.1172/JCI19552.
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Perspective Series

The potential for gene repair via triple helix formation

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Abstract

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.

Authors

Michael M. Seidman, Peter M. Glazer

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