National Institute of Pharmaceutical Education and Research (NIPER), Department of Biotechnology, Sector 67, SAS. Nagar, 160 062 Punjab, India +91 172 2214 682 ; +91 172 2214 692 ;
Expert Opin Drug Discov. 2008 Oct;3(10):1189-207. doi: 10.1517/17460441.3.10.1189.
With the advent of nucleic acids as therapeutic molecules, the traditional drug discovery and development process has undergone a radical change. Because nucleic acids target complementary sequences and the recognition is on the basis of Watson-Crick base pair formation, the specificity of the whole process is high. The unwanted side reactions, which are a common phenomenon observed with small molecules, are thus cut down.
The objective of this review is to look at the concept of antisense oligonucleotides as nucleic acid medicines and trace the history of drug molecules based on this concept that are at various stages of advancement. The problems encountered in the development process and the possible delivery routes are critically analyzed.
Although specificity and selectivity are the key features of antisense oligonucleotides, the need to target the right tissues and reach the nucleus remains a challenge to overcome.
随着核酸作为治疗分子的出现，传统的药物发现和开发过程发生了根本性的变化。由于核酸靶向互补序列，并且识别是基于沃森-克里克碱基对形成的，因此整个过程的特异性很高。因此，小分子中常见的不必要的副作用减少了。
本综述的目的是探讨反义寡核苷酸作为核酸药物的概念，并追溯基于该概念的处于不同进展阶段的药物分子的历史。批判性地分析了在开发过程中遇到的问题和可能的给药途径。
尽管特异性和选择性是反义寡核苷酸的关键特征，但靶向正确的组织并进入细胞核仍然是一个需要克服的挑战。
