Therapeutic Aptamers: Evolving to Find their Clinical Niche.
Therapeutic Aptamers: Evolving to Find their Clinical Niche.
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DOI：
10.2174/0929867326666191001125101
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发表时间：
2020
影响因子：
4.1
通讯作者：
Sullenger BA
中科院分区：
文献类型：
--
作者：
Nimjee SM;Sullenger BA
短的寡核苷酸，称为适体，可以折叠成三维结构，使它们能够选择性地结合和抑制致病蛋白的活性，这一发现已经超过25年了。SELEX方法学的发明预示着这样一个时代的到来，在这个时代中，可以针对各种各样的治疗靶点产生这种基于核酸的配体。大量的适体现在已经在实验室中通过组合化学方法鉴定，而且，越来越多的适体已经在自然界中被发现。这种适体的亲和力和活性经常与抗体的亲和力和活性进行比较，但与大量且不断增加的治疗性抗体相比，这些试剂中只有少数进入临床研究。一种靶向VEGF的治疗性适体已经上市，而另外3种适体已经进入III期临床试验。在这篇文章中，我们希望读者能够理解，适体成为一类药物的成功与核酸生物化学无关，而更多地与靶点验证和整体药物化学有关。
The discovery that short oligonucleotides, termed aptamers, can fold into three-dimensional structures that allow them to selectively bind and inhibit the activity of pathogenic proteins is now over 25 years old. The invention of the SELEX methodology heralded in an era in which such nucleic acid-based ligands could be generated against a wide variety of therapeutic targets. A large number of aptamers have now been identified by combinatorial chemistry methods in the laboratory and moreover, an increasing number have been discovered in nature. The affinities and activities of such aptamers have often been compared to that of antibodies, yet only a few of these agents have made it into clinical studies compared to a large and increasing number of therapeutic antibodies. One therapeutic aptamer targeting VEGF has made it to market, while 3 others have advanced as far as phase III clinical trials. In this manuscript, we hope the reader appreciates that the success of aptamers becoming a class of drugs is less about nucleic acid biochemistry and more about target validation and overall drug chemistry.
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影响因子：
37.8
作者：
Gilbert, James C.;DeFeo-Fraulini, Tia;Schaub, Robert G.
通讯作者：
Schaub, Robert G.
影响因子：
158.5
作者：
Gragoudas, ES;Adamis, AP;Guyer, DR
通讯作者：
Guyer, DR
影响因子：
1
作者：
Dunn, Evan N.;Sheth, Veeral S.;Hariprasad, Seenu M.
通讯作者：
Hariprasad, Seenu M.
影响因子：
4.8
作者：
BEIGELMAN, L;MCSWIGGEN, JA;USMAN, N
通讯作者：
USMAN, N
在分子支架上组装OX40适体，以创建受体激活的适体。
影响因子：
--
作者：
Dollins, Claudia M.;Nair, Smita;Boczkowski, David;Lee, Jaewoo;Layzer, Juliana M.;Gilboa, Eli;Sullenger, Bruce A.
通讯作者：
Sullenger, Bruce A.