Novel antisense and peptide nucleic acid strategies for controlling gene expression

Novel antisense and peptide nucleic acid strategies for controlling gene expression
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DOI:
10.1021/bi0122112
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发表时间:
2002-04-09
期刊:
影响因子:
2.9
通讯作者:
Corey, DR
Corey, DR
中科院分区:
生物学3区
文献类型:
--
作者:
Braasch, DA;Corey, DR

文献摘要

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反义寡核苷酸有可能为基础科学和医学做出革命性的贡献。寡核苷酸可以结合 mRNA 并抑制翻译。由于它们可以快速合成以与任何序列互补,因此它们为利用现有的大量基因组信息提供了理想的工具。然而,直到最近,这种潜力很大程度上还只是理论上的,反义实验常常产生不确定或误导性的结果。本综述将讨论现有的一些不同类型寡聚物的化学和生物特性,并描述寡核苷酸在体外和体内使用所面临的挑战。具有改进化学性质的低聚物,结合细胞生物学的进步和临床试验的成功,为基础研究、生物技术和医学提供了强大的新选择。
Antisense oligonucleotides have the potential to make revolutionary contributions to basic science and medicine. Oligonucleotides can bind mRNA and inhibit translation. Because they can be rapidly synthesized to be complementary to any sequence, they offer ideal tools for exploiting the massive amount of genome information now available. However, until recently, this potential was largely theoretical, and antisense experiments often produced inconclusive or misleading outcomes. This review will discuss the chemical and biological properties of some of the different types of oligomers now available and describe the challenges confronting in vitro and in vivo use of oligonucleotides. Oligomers with improved chemical properties, combined with advances in cell biology and success in clinical trials, are affording powerful new options for basic research, biotechnology, and medicine.