Recent advances in mRNA-based therapeutics for neurodegenerative diseases and brain tumors

Messenger RNA (mRNA) therapy is an innovative approach that delivers specific protein-coding information. By promoting the ribosomal synthesis of target proteins within cells, it supplements functional or antigenic proteins to treat diseases. Unlike traditional gene therapy, mRNA does not need to enter the cell nucleus, reducing the risks associated with gene integration. Moreover, protein expression levels can be regulated by adjusting the dosage and degradation rates of mRNA. As a new generation gene therapy strategy, mRNA therapy represents the latest advancements and trends in the field. It offers advantages such as precision, safety, and ease of modification. It has been widely used in the prevention of COVID-19. Unlike acute conditions such as cerebral hemorrhage and stroke that often require immediate surgical or interventional treatments, neurodegenerative diseases (NDs) and brain tumors progress relatively slowly and face challenges such as the blood–brain barrier and complex pathogenesis. These characteristics make them particularly suitable for mRNA therapy. With continued research, mRNA-based therapeutics are expected to play a significant role in the prevention and treatment of NDs and brain tumors. This paper reviews the preparation and delivery of mRNA drugs and summarizes the research progress of mRNA gene therapy in treating NDs and brain tumors. It also discusses the current challenges, providing a theoretical basis and reference for future research in this field.