Department of Medicine, University of Massachusetts Chan Medical School, Mattapan, MA 02126, USA.
MassBiologics of the University of Massachusetts Chan Medical School, Mattapan, MA 02126, USA.
Curr Org Synth. 2024;21(4):436-455. doi: 10.2174/1570179420666230502123950.
This article covers the triazole-linked nucleic acids where the triazole linkage (TL) replaces the natural phosphate backbone. The replacement is done at either a few selected linkages or all the phosphate linkages. Two triazole linkages, the four-atom TL1 and the six-atom TL2, have been discussed in detail. These triazole-modified oligonucleotides have found a wide range of applications, from therapeutics to synthetic biology. For example, the triazole-linked oligonucleotides have been used in the antisense oligonucleotide (ASO), small interfering RNA (siRNA) and clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 technology as therapeutic agents. Due to the ease of the synthesis and a wide range of biocompatibility, the triazole linkage TL2 has been used to assemble a functional 300-mer DNA from alkyne- and azide-functionalized 100-mer oligonucleotides as well as an epigenetically modified variant of a 335 base-pair gene from ten short oligonucleotides. These outcomes highlight the potential of triazole-linked nucleic acids and open the doors for other TL designs and artificial backbones to fully exploit the vast potential of artificial nucleic acids in therapeutics, synthetic biology and biotechnology.
本文涵盖了三唑连接的核酸,其中三唑键(TL)取代了天然的磷酸骨架。这种取代可以在少数选定的连接点或所有磷酸连接点上进行。本文详细讨论了两种三唑连接,即四原子 TL1 和六原子 TL2。这些三唑修饰的寡核苷酸已经在治疗学和合成生物学等领域得到了广泛的应用。例如,三唑连接的寡核苷酸已被用作反义寡核苷酸(ASO)、小干扰 RNA(siRNA)和簇状规则间隔短回文重复(CRISPR)-Cas9 技术的治疗剂。由于合成的简便性和广泛的生物相容性,TL2 已被用于从炔基和叠氮基官能化的 100 -mer 寡核苷酸组装功能性 300-mer DNA,以及从十个短寡核苷酸组装 335 碱基对基因的表观遗传修饰变体。这些结果突出了三唑连接核酸的潜力,并为其他 TL 设计和人工骨架打开了大门,以充分利用人工核酸在治疗学、合成生物学和生物技术中的巨大潜力。