Uil Taco G, Haisma Hidde J, Rots Marianne G
Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands.
Nucleic Acids Res. 2003 Nov 1;31(21):6064-78. doi: 10.1093/nar/gkg815.
Designer molecules that can specifically target pre-determined DNA sequences provide a means to modulate endogenous gene function. Different classes of sequence-specific DNA-binding agents have been developed, including triplex-forming molecules, synthetic polyamides and designer zinc finger proteins. These different types of designer molecules with their different principles of engineered sequence specificity are reviewed in this paper. Furthermore, we explore and discuss the potential of these molecules as therapeutic modulators of endogenous gene function, focusing on modulation by stable gene modification and by regulation of gene transcription.
能够特异性靶向预定DNA序列的设计分子为调节内源性基因功能提供了一种手段。人们已经开发出不同类型的序列特异性DNA结合剂，包括三链形成分子、合成聚酰胺和设计锌指蛋白。本文综述了这些具有不同工程化序列特异性原理的不同类型设计分子。此外，我们探讨并讨论了这些分子作为内源性基因功能治疗调节剂的潜力，重点关注通过稳定基因修饰和基因转录调控进行的调节。
