AM Biotechnologies LLC, Houston, TX 77034, USA.
Expert Opin Drug Discov. 2011 Jan;6(1):75-87. doi: 10.1517/17460441.2011.537321.
Therapeutic aptamers are synthetic, structured oligonucleotides that bind to a very broad range of targets with high affinity and specificity. They are an emerging class of targeting ligand that show great promise for treating a number of diseases. A series of aptamers currently in various stages of clinical development highlights the potential of aptamers for therapeutic applications.
This review covers in vitro selection of oligonucleotide ligands, called aptamers, from a combinatorial library using the Systematic Evolution of Ligands by Exponential Enrichment process as well as the other known strategies for finding aptamers against various targets.
Readers will gain an understanding of the highly useful strategies for successful aptamer discovery. They may also be able to combine two or more of the presented strategies for their aptamer discovery projects.
Although many processes are available for discovering aptamers, it is not easy to discover an aptamer candidate that is ready to move toward pharmaceutical drug development. It is also apparent that there have been relatively few therapeutic advances and clinical trials undertaken due to the small number of companies that participate in aptamer development.
治疗性适体是合成的、结构化的寡核苷酸,能以高亲和力和特异性结合到非常广泛的靶标上。它们是一类新兴的靶向配体,在治疗多种疾病方面显示出巨大的潜力。目前有一系列适体处于不同的临床开发阶段,这突出了适体在治疗应用中的潜力。
使用指数富集的配体系统进化(SELEX)过程从组合文库中体外选择寡核苷酸配体(称为适体),以及针对各种靶标寻找适体的其他已知策略。
读者将了解到成功发现适体的高度有用的策略。他们也可以将两个或更多呈现的策略结合到他们的适体发现项目中。
尽管有许多过程可用于发现适体,但要发现一个准备好进入药物开发的适体候选物并不容易。由于参与适体开发的公司相对较少,因此在治疗性进展和临床试验方面也相对较少,这一点也很明显。