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Current landscape of mRNA technologies and delivery systems for new modality therapeutics

Ruei‐Min LuBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanHsiang-En HsuBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanSer John Lynon P. PerezBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanMonika KumariInstitute of Cellular and Organismic Biology, Academia Sinica, No. 128, Academia Road, Section 2, Taipei, 11529, TaiwanGuanhong ChenBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanMing-Hsiang HongBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanYin-Shiou LinBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanChing-Hang LiuBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanShih-Han KoBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanChristian Angelo P. ConcioInstitute of Chemistry, Academia Sinica, No. 128, Academia Road, Section 2, Taipei, 11529, TaiwanYi‐Jen SuInstitute of Cellular and Organismic Biology, Academia Sinica, No. 128, Academia Road, Section 2, Taipei, 11529, TaiwanYi‐Han ChangBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, TaiwanWen‐Shan LiBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, Taiwan. [email protected]Han‐Chung WuBiomedical Translation Research Center, Academia Sinica, Taipei, 11571, Taiwan. [email protected]
2024en
ABI

Аннотация

Realizing the immense clinical potential of mRNA-based drugs will require continued development of methods to safely deliver the bioactive agents with high efficiency and without triggering side effects. In this regard, lipid nanoparticles have been successfully utilized to improve mRNA delivery and protect the cargo from extracellular degradation. Encapsulation in lipid nanoparticles was an essential factor in the successful clinical application of mRNA vaccines, which conclusively demonstrated the technology's potential to yield approved medicines. In this review, we begin by describing current advances in mRNA modifications, design of novel lipids and development of lipid nanoparticle components for mRNA-based drugs. Then, we summarize key points pertaining to preclinical and clinical development of mRNA therapeutics. Finally, we cover topics related to targeted delivery systems, including endosomal escape and targeting of immune cells, tumors and organs for use with mRNA vaccines and new treatment modalities for human diseases.

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