The potential for gene repair via triple helix formation.
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.
Seidman
Michael M
MM
Department of Therapeutic Radiology, Yale University School of Medicine, P.O. Box 208040, New Haven, Connecticut 06520-8040, USA.
Glazer
Peter M
PM
D000818:Animals; D004247:DNA / Q000378:metabolism; D004260:DNA Repair; D005821:Genetic Techniques; D015316:Genetic Therapy; D008956:Models, Chemical; D011995:Recombination, Genetic
C051802:triplex DNA; D004247:DNA