Kaihatsu Kunihiro, Janowski Bethany A, Corey David R
Departments of Pharmacology and Biochemistry, University of Texas Southwestern Medical Center at Dallas, 5323 Harry Hines Boulevard, Dallas, TX 75390 USA.
Chem Biol. 2004 Jun;11(6):749-58. doi: 10.1016/j.chembiol.2003.09.014.
The recognition of cellular nucleic acids by synthetic oligonucleotides is a versatile strategy for regulating biological processes. The vast majority of published studies have focused on antisense oligonucleotides that target mRNA, but it is also possible to design antigene oligonucleotides that are complementary to chromosomal DNA. Antigene oligomers could be used to inhibit the expression of any gene or analyze promoter structure and the mechanisms governing gene regulation. Other potential applications of antigene oligomers include activation of expression of chosen genes or the introduction of mutations to correct genetic disease. Peptide nucleic acid (PNA) is a nonionic DNA/RNA mimic that possesses outstanding potential for recognition of duplex DNA. Here we describe properties of PNAs and the challenges for their development as robust antigene agents.
通过合成寡核苷酸识别细胞核酸是调节生物过程的一种通用策略。绝大多数已发表的研究都集中在靶向mRNA的反义寡核苷酸上，但也可以设计与染色体DNA互补的反基因寡核苷酸。反基因寡聚物可用于抑制任何基因的表达或分析启动子结构以及基因调控机制。反基因寡聚物的其他潜在应用包括激活所选基因的表达或引入突变以纠正遗传疾病。肽核酸（PNA）是一种非离子型DNA/RNA模拟物，在识别双链DNA方面具有出色的潜力。在这里，我们描述了PNA的特性及其作为强大的反基因药物开发所面临的挑战。
