RNA plays a central role in the expression of all genes. Because any sequence within RNA can be recognized by complementary base pairing, synthetic oligonucleotides and oligonucleotide mimics offer a general strategy for controlling processes that affect disease. The two primary antisense approaches for regulating expression through recognition of cellular RNAs are single-stranded antisense oligonucleotides and duplex RNAs. This review will discuss the chemical modifications and molecular mechanisms that make synthetic nucleic acid drugs possible. Lessons learned from recent clinical trials will be summarized. Ongoing clinical trials are likely to decisively test the adequacy of our current generation of antisense nucleic acid technologies and highlight areas where more basic research is needed.

中文翻译：

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反义寡核苷酸和双链体RNA的化学，机理和临床状态

RNA在所有基因的表达中起着核心作用。由于RNA内的任何序列均可被互补碱基配对识别，因此合成的寡核苷酸和寡核苷酸模拟物为控制影响疾病的过程提供了一般策略。通过识别细胞RNA调节表达的两种主要反义方法是单链反义寡核苷酸和双链RNA。这篇综述将讨论使合成核酸药物成为可能的化学修饰和分子机理。从最近的临床试验中学到的经验教训将被总结。正在进行的临床试验可能会决定性地测试我们当前这一代反义核酸技术的适用性，并突出显示需要更多基础研究的领域。