Wyeth-Ayerst Research, Philadelphia, PA 19101, USA.
Trends Cardiovasc Med. 1994 Mar-Apr;4(2):51-5. doi: 10.1016/1050-1738(94)90009-4.
Antisense oligonucleotides have great potential as rationally designed therapeutic drugs by taking advantage of the basic Watson-Crick base pairing of nucleic acids. Such oligonucleotides may block synthesis of a specific protein, such a c-myb, and prevent smooth muscle proliferation involved in restenosis. Although promising, the technology has some major hurdles to overcome before fruition. These include obtaining a stable backbone that can enter cells readily, overcoming problems of chirality, and solving the problems of delivery and metabolism. Although there are many reports of successful experiments using antisense oligonucleotides, one must always keep in mind the complex nature of these experiments, as well as nonspecific effects that may masquerade as antisense effects.
反义寡核苷酸通过利用核酸的基本沃森-克里克碱基配对，具有作为合理设计的治疗药物的巨大潜力。这些寡核苷酸可以阻断特定蛋白质（如 c-myb）的合成，并防止涉及再狭窄的平滑肌增殖。尽管有前途，但该技术在取得成果之前还有一些重大障碍需要克服。这些包括获得能够轻易进入细胞的稳定骨架，克服手性问题，并解决传递和代谢问题。尽管有许多成功使用反义寡核苷酸进行实验的报道，但人们必须始终牢记这些实验的复杂性，以及可能伪装为反义效应的非特异性效应。
