Mojzísek Martin
Charles University in Prague, Faculty of Medicine in Hradec Králové, Czech Republic: Department of Medical Biology and Genetics.
Acta Medica (Hradec Kralove). 2004;47(3):151-6.
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.
本综述探讨了反基因策略，即寡核苷酸与双螺旋DNA的大沟或小沟结合，在那里形成局部三链螺旋。本文主要关注的是三链形成寡核苷酸（TFO）。这些是短的合成单链DNA，它们以序列特异性方式识别双链DNA中的聚嘌呤：聚嘧啶区域并形成三链体。因此，本文讨论了通过三链螺旋形成识别DNA的机制，以及TFO的主要特征，并强调了仍有待克服的主要障碍。TFO可以在转录水平上选择性抑制基因表达，或通过对人类细胞进行直接基因组修饰来修复遗传缺陷。这些特性使TFO成为基因修复和/或表达调控的潜在有力治疗工具。
