Lundstrom Kenneth
PanTherapeutics, Route de Lavaux 49, CH1095 Lutry, Switzerland.
Future Sci OA. 2018 May 4;4(5):FSO300. doi: 10.4155/fsoa-2017-0151. eCollection 2018 Jun.
Drugs and vaccines based on mRNA and RNA viruses show great potential and direct translation in the cytoplasm eliminates chromosomal integration. Limitations are associated with delivery and stability issues related to RNA degradation. Clinical trials on RNA-based drugs have been conducted in various disease areas. Likewise, RNA-based vaccines for viral infections and various cancers have been subjected to preclinical and clinical studies. RNA delivery and stability improvements include RNA structure modifications, targeting dendritic cells and employing self-amplifying RNA. Single-stranded RNA viruses possess self-amplifying RNA, which can provide extreme RNA replication in the cytoplasm to support RNA-based drug and vaccine development. Although oligonucleotide-based approaches have demonstrated potential, the focus here is on mRNA- and RNA virus-based methods.
基于信使核糖核酸（mRNA）和核糖核酸（RNA）病毒的药物和疫苗显示出巨大潜力，且在细胞质中的直接翻译消除了染色体整合。局限性与RNA降解相关的递送和稳定性问题有关。基于RNA的药物已在多个疾病领域进行了临床试验。同样，用于病毒感染和各种癌症的基于RNA的疫苗也已进行了临床前和临床研究。RNA递送和稳定性的改进包括RNA结构修饰、靶向树突状细胞以及使用自我扩增RNA。单链RNA病毒拥有自我扩增RNA，其可在细胞质中实现极高的RNA复制，以支持基于RNA的药物和疫苗开发。尽管基于寡核苷酸的方法已显示出潜力，但本文重点关注基于mRNA和RNA病毒的方法。
