Universidade de São Paulo, Instituto de Quimica, Departamento de Bioquímica, São Paulo, SP, CEP 05508-900, Brazil.
Expert Opin Ther Pat. 2009 Nov;19(11):1603-13. doi: 10.1517/13543770903313746.
RNA and DNA aptamers recognize their targets with high specificity and affinity. These aptamers can be developed against almost any target protein through iterative cycles of in vitro screening of a combinatorial oligonucleotide library for target binding. Aptamer sequences from the final pool of in vitro selection are screened for pharmacological activity and possible medical applications.
Chemical modifications and improvements of the identification of aptamer selection procedures made aptamers rival antibodies in diagnostic and therapeutic applications. This article reviews recent literature and patents and discusses the properties of aptamers as high-affinity and specificity target binders as well as their stability in biological fluids that turns them into therapeutic agents.
The development of aptamers into compounds with therapeutic and diagnostic compounds has resulted in patents protecting the sequences and the use of these oligonucleotides. Several of these patented aptamers are currently being tested in Phase I or II clinical trials. Moreover, an anti-VEGF aptamer has already been approved by the FDA for treatment of age-related macular degeneration in humans.
RNA 和 DNA 适体以高特异性和亲和力识别其靶标。这些适体可以通过体外筛选组合寡核苷酸文库与靶标结合来针对几乎任何靶标蛋白进行开发。从最终的体外选择池中筛选适体序列，以评估其药理学活性和可能的医学应用。
通过对适体选择程序的鉴定进行化学修饰和改进，使适体在诊断和治疗应用中与抗体相媲美。本文综述了最近的文献和专利，并讨论了适体作为高亲和力和特异性靶标结合物的特性，以及它们在生物流体中的稳定性，使它们成为治疗剂。
将适体开发成具有治疗和诊断作用的化合物已导致专利保护这些寡核苷酸的序列和用途。目前正在进行临床试验 I 期或 II 期研究的专利适体有几种。此外，一种抗 VEGF 适体已被 FDA 批准用于治疗人类年龄相关性黄斑变性。
