Research Advances in mRNA Therapeutics
ZHU Liuyu, WU Junjun*
Benefiting from breakthroughs in nucleoside modification and lipid nanoparticle delivery systems,mRNA therapeutics have developed rapidly in recent years and have emerged as an important technological approach in thebiopharmaceutical field. The successful application of mRNA vaccines during the SARS-CoV-2 infection pandemic furtherdemonstrated their potential for rapid development and large-scale deployment. This review summarizes recent advancesin three major application areas of mRNA therapeutics, including vaccines, protein replacement therapy, and gene editing.Linear mRNA remains the predominant approach for mRNA vaccine development, while circRNA (circular RNA) showspromising potential owing to its enhanced stability and prolonged protein expression. Meanwhile, mRNA-mediated proteinreplacement therapy has provided new therapeutic strategies for inherited metabolic disorders. In the field of gene editing,mRNA-based delivery of CRISPR editing systems has attracted considerable attention owing to its favorable safety profile.Despite the significant advances in mRNA therapeutics, their clinical translation remains hindered by key challenges, including insufficient tissue-specific delivery efficiency. With continued advances in RNA engineering and delivery optimization,mRNA therapeutics are expected to become an important common technological foundation that connects vaccine development, protein replacement therapy and gene editing.
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