Acta Med. 2004, 47: 151-156

Triplex Forming Oligonucleotides – Tool for Gene Targeting

Martin Mojžíšek

Charles University in Prague, Faculty of Medicine in Hradec Králové, Department of Medical Biology and Genetics, Hradec Králové, Czech Republic

Received March 1, 2004
Accepted May 1, 2004

This review deals with the antigene strategy whereby an oligonucleotide binds to the major or minor groove of double helical DNA where it forms a local triple helix. Preoccupation of this article is triplex-forming oligonucleotides (TFO). These are short, synthetic single-stranded DNAs that recognize polypurine:polypyrimidine regions in double stranded DNA in a sequence-specific manner and form triplex. Therefore, the mechanisms for DNA recognition by triple helix formation are discussed, together with main characteristics of TFO and also major obstacles that remain to be overcome are highlighted. TFOs can selectively inhibit gene expression at the transcriptional level or repair genetic defect by direct genome modification in human cells. These qualities makes TFO potentially powerful therapeutic tool for gene repair and/or expression regulation.


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