mRNA疗法的研究进展与挑战
摘要
<p indent="0mm">mRNA-based therapeutics are widely used for treating various diseases and represent an emerging preventive and therapeutic approach that can be an effective alternative to DNA- and recombinant protein-based therapeutics. Accompanied by the continuous development of <italic>in vitro</italic> synthesis, purification, modification, and delivery system optimization, mRNA-based therapeutics have been substantially improved in terms of stability, translation efficiency, and controllable immunogenicity. At present, mRNA-based drugs are receiving increasing attention in cancer therapy, infectious disease prevention, protein replacement, and gene editing. Moreover, low costs and fast turnover rates in large-scale production confer great potential for the development of mRNA therapeutic platforms. This paper reviews the current status of mRNA-based therapeutics used for various diseases in both preclinical and clinical stages. It also provides a reasonable discussion on key barriers in this field and an outlook towards its future.