Nishikawa Makiya
Department of Biopharmaceutics and Drug Metabolism, Graduate School of Pharmaceutical Sciences, Kyoto University.
Drug Discov Ther. 2016 Nov 28;10(5):271-272. doi: 10.5582/ddt.2016.01056. Epub 2016 Oct 23.
Nucleic acids are biologically active materials, and chemically modified nucleic acids are now being used in nucleic acid drugs. DNA, one of the two types of nucleic acids, can also be used as a material to construct DNA-based delivery systems, such as DNA hydrogel, for therapeutic compounds. Use of chemically modified nucleic acids would greatly increase the therapeutic potency of such DNA-based delivery systems. However, attention should be paid to the differences in the physicochemical properties of natural and chemically modified nucleic acids. Another, more important concern for chemically modified nucleic acids is the high cost of their synthesis. Reducing the cost of synthesizing oligonucleotides, and especially ones with chemically modified nucleic acids, is crucial to the expanded use of both nucleic acid drugs and DNA-based delivery systems.
核酸是具有生物活性的物质,化学修饰的核酸目前正被用于核酸药物中。核酸的两种类型之一——DNA,也可以用作构建基于DNA的递送系统的材料,如用于治疗性化合物的DNA水凝胶。使用化学修饰的核酸将大大提高此类基于DNA的递送系统的治疗效力。然而,应注意天然核酸和化学修饰核酸在物理化学性质上的差异。对于化学修饰核酸,另一个更重要的问题是其合成成本高昂。降低寡核苷酸尤其是化学修饰核酸寡核苷酸的合成成本,对于核酸药物和基于DNA的递送系统的广泛应用至关重要。