Over the past several decades, the concept of using molecules composed partially or wholly of nucleic acids as therapeutic moieties, and the modification of such molecules via synthetic strategies, has been discussed and actively pursued by many academic and industrial laboratories. As compared to small molecules (and more recently antibodies and other protein-based drugs) the use of oligonucleotides and related compounds as therapeutics has advanced more slowly—a fact that is not surprising, given the challenges that such molecules (and their investigators) face. Nucleic acids are large, highly charged, rapidly degraded and cleared from the body, and offer generally poor pharmacological properties. The development of nucleic acids as potential therapeutic agents has nonetheless moved forward at a steady pace, owing in large part to important discoveries regarding their role in regulating gene expression, and in part to the development of increasingly sophisticated synthetic and biochemical methods to alter many of the physical properties that might limit their potential as drugs.

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社论：核酸研究与核酸治疗学

在过去的几十年中，许多学术和工业实验室已经讨论并积极地追求使用部分或全部由核酸组成的分子作为治疗部分以及通过合成策略对此类分子进行修饰的概念。与小分子（以及最近的抗体和其他基于蛋白质的药物）相比，使用寡核苷酸和相关化合物作为治疗剂的进展更为缓慢-考虑到此类分子（及其研究人员）面临的挑战，这一事实不足为奇。核酸大，电荷高，迅速降解并从体内清除，并且通常具有较差的药理特性。尽管如此，核酸作为潜在治疗剂的发展仍在稳步推进，