mRNA治疗用纳米载体研究新进展  

New progress in the research of nanocarriers for mRNA-based therapeutics

作　　者：石立旺 张楠 祝纶宇[1] 王颖 郭敏[1] 杜超[1] SHI Li-wang;ZHANG Nan;ZHU Lun-yu;WANG Ying;GUO Min;DU Chao

机构地区：[1]中石化(北京)化工研究院有限公司医用卫生材料研究所,北京100013

出　　处：《中国医药生物技术》2024年第1期45-51,56,共8页Chinese Medicinal Biotechnology

摘　　要：mRNA是一种单链核糖核酸,由一条DNA转录而成,携带蛋白质合成的编码信息,进一步转录并加工成功能蛋白。在基因工程的协同下,合成的mRNA在结构上类似于天然mRNA,使患者能够在自己的身体中产生治疗性蛋白质,从而达到预防或治疗疾病的目的。mRNA治疗避免了其遗传物质整合宿主基因组的风险,也不需要进入细胞核进行转染,它甚至可以在不分裂的细胞中表达。因此,基于mRNA的基因疗法成为极具潜力的临床治疗手段。高效、安全地递送mRNA是其临床应用的两个主要制约因素。目前虽然已有很多报道通过修饰mRNA的结构来提高其稳定性和耐受性,但mRNA的递送效果仍然有待提高。近年来,纳米生物技术取得的重大进展为mRNA纳米载体开发提供了技术支撑。纳米载体能够克服递送过程中的生理障碍,使mRNA到达目标部位。本文介绍了纳米给药系统的设计原则和研究热点。重点聚焦新兴纳米载体在mRNA传递中发挥的作用以及在递送功能研究方面的最新进展;最后,对mRNA的纳米载体递送技术发展前景进行了展望。mRNA is a type of single-stranded ribonucleic acid that is transcribed from a single strand of DNA and carries the encoding information for protein synthesis,which is further transcribed and processed into functional proteins.In coordination with genetic engineering,the synthesized mRNA is structurally similar to natural mRNA,enabling patients to produce therapeutic proteins in their own bodies,thus achieving the purpose of preventing or treating disease.mRNA does not have the risk of being incorporated into the host genome and does not need to enter the nucleus for transfection.Moreover,it can even be expressed in nondividing cells.Therefore,mRNA-based therapeutics have become a promising clinical treatment strategy.The efficiency and safety delivery of mRNA remain to be crucial constraints for clinical application of the mRNA therapeutics.Although there have been many reports about how to improve the stability and tolerability of mRNA by directly retouching the mRNA structure,there is still an urgent need to improve the delivery of mRNA.Recently,significant progress has been made in nanobiotechnology,providing technical supports for developing mRNA nanocarriers.The nanocarriers can overcome the physiological barriers in the delivery process and enable the mRNA to reach the target site.In this review,the design principles and research hotspots of nano drug delivery system are introduced.Emphasis will be placed on the role of mRNA delivery in emerging nanomaterials,cell-derived exosomes,and recent advances in delivery functions.Finally,the development prospects of mRNA nanomaterials delivery technology are prospected.

关 键 词：外泌体 基因治疗 mRNA递送 纳米载体 

分 类 号：R943[医药卫生—药剂学]