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信使 RNA 疗法:现代医学中的进展、挑战与未来展望

Curr Pharm Des · 2026年9月25日 · Sharma 等 6 位作者

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一分钟了解要点综述 mRNA 疗法的技术进展、递送与知识产权挑战,展望其从应急工具走向常规治疗。

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The rapid clinical validation of messenger RNA (RNA) during the COVID-19 pandemic has catalyzed a shift from traditional vaccine development toward a versatile, programmable therapeutic platform. This review provides a comprehensive analysis of the mRNA landscape, moving beyond historical milestones to critically evaluate the structural biology and synthetic modifications required for therapeutic stabilization. We examine the transition from Lipid Nanoparticle (LNP) dominance toward next-generation delivery vehicles designed to overcome current limitations in tissue targeting and systemic reactogenicity. Furthermore, this article contrasts mRNA with DNA-based modalities, specifically focusing on the safety profiles and rapid design cycles that favor mRNA in acute and personalized indications. A significant portion of this synthesis is dedicated to the shifting intellectual property landscape, interpreting how patent concentration among key industry players influences global innovation and market accessibility. By identifying current gaps in manufacturing scalability and regulatory harmonization, this review previews the transition of mRNA technology from an emergency response tool to a mainstay of chronic disease management and precision oncology. Overall, this work outlines the technical and socio-economic hurdles that must be cleared to realize a new era of adaptable, "on-demand" healthcare solutions.

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