纳米材料在基因治疗中的研究进展:从肿瘤治疗到新冠病毒疫苗

Nanomaterials in gene therapy from tumor therapy to novel coronavirus vaccine

基因治疗作为一种精准有效的策略,可用于治疗包括癌症在内的多种疾病以及预防病毒性感染疾病.然而,基因治疗中使用的核酸药物自身不稳定性和大尺寸阻碍了它们的广泛应用.纳米材料因其免疫原性低、可控性好、易于进行表面修饰等特点,已被证明是基因治疗中最有前途的载体之一.特别是新型冠状病毒肺炎(COVID-19)疫情爆发以来,与传统疫苗不同的mRNA疫苗备受关注,而脂质纳米颗粒(LNP)作为该疫苗的递送系统发挥了至关重要的作用,展现了纳米材料巨大的应用前景.本文概述了基因治疗的主要类型;介绍了基于脂质的纳米颗粒、基于聚合物的纳米颗粒、无机纳米颗粒及新型纳米材料如碳点等核酸递送平台;强调了其在肿瘤治疗和COVID-19核酸疫苗中的最新研究进展;提出了纳米材料用于基因治疗的挑战与前景.

Gene therapy,a precise and effective therapeutic strategy,can be applied to a variety of diseases including cancer and infectious diseases.However,instability and large size of nucleic acid drugs used in gene therapy hinder their effective applications.Nanomaterials have proved to be the most promising in clinical tumor gene therapy due to low immunogenicity,good controllability,easy surface modification and multifunctioning.In particular,mRNA vaccines have attracted much attention since the outbreak of COVID-19.Lipid nanoparticles(LNP)has been used successfully as the delivery system of this vaccine.Major types of gene therapy were outlined in this minireview.The delivery platform for nucleic acids could be based on lipids,cationic polymers,inorganic nanoparticles and other new nanomaterials such as carbon dots.Their applications in tumor therapy and development of novel coronavirus vaccine are emphasized.Future challenges and prospects of nanomaterial for gene therapy are presented.