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

被引：14

作者：

Lu, Ruei-Min ^{[1
]}

Hsu, Hsiang-En ^{[1
]}

Perez, Ser John Lynon P. ^{[1
]}

Kumari, Monika ^{[2
]}

Chen, Guan-Hong ^{[1
]}

Hong, Ming-Hsiang ^{[1
]}

Lin, Yin-Shiou ^{[1
]}

Liu, Ching-Hang ^{[1
]}

Ko, Shih-Han ^{[1
]}

Concio, Christian Angelo P. ^{[3
]}

Su, Yi-Jen ^{[2
]}

Chang, Yi-Han ^{[1
]}

Li, Wen-Shan ^{[1
,3
]}

Wu, Han-Chung ^{[1
,2
]}

机构：

[1] Acad Sinica, Biomed Translat Res Ctr, Taipei 11571, Taiwan

[2] Acad Sinica, Inst Cellular & Organism Biol, 128 Acad Rd,Sect 2, Taipei 11529, Taiwan

[3] Acad Sinica, Inst Chem, 128 Acad Rd,Sect 2, Taipei 11529, Taiwan

来源：

JOURNAL OF BIOMEDICAL SCIENCE | 2024年 / 31卷 / 01期

关键词：

mRNA Technology; mRNA therapeutics; Lipid nanoparticles; Targeting mRNA delivery systems; New modality therapeutics; CHIMERIC ANTIGEN RECEPTOR; LIPID NANOPARTICLES; IN-VIVO; PHOSPHODIESTER BONDS; GENE; PURIFICATION; CELLS; EXPRESSION; CANCER; PSEUDOURIDINE;

D O I：

10.1186/s12929-024-01080-z

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

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.

引用

页数：36

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