生物技术进展 ›› 2024, Vol. 14 ›› Issue (1): 1-16.DOI: 10.19586/j.2095-2341.2023.0139

• 生物制品研发与技术专题 •    

mRNA药物的结构和临床应用

常东峰(), 孙召朋()   

  1. 石药集团巨石生物有限责任公司,石家庄 050015
  • 收稿日期:2023-10-26 接受日期:2023-11-28 出版日期:2024-01-25 发布日期:2024-02-05
  • 通讯作者: 孙召朋
  • 作者简介:常东峰 E-mail: changdongfeng@cspc.cn
  • 基金资助:
    河北省生物医药创新专项(22372405D)

Structure and Clinical Application of mRNA Drugs

Dongfeng CHANG(), Zhaopeng SUN()   

  1. CSPC Megalith Biopharmaceutical Co. ,Ltd. ,Shijiazhuang 050015,China
  • Received:2023-10-26 Accepted:2023-11-28 Online:2024-01-25 Published:2024-02-05
  • Contact: Zhaopeng SUN

摘要:

mRNA凭借其有效性、安全性和易大规模生产等特点,在预防急性传染病方面显示出巨大的潜力。mRNA代表了一个新兴的精准医学领域,几种针对传染病和癌症等疗法的mRNA在体内和体外都显示出良好效果。mRNA稳定性好、免疫原性高、不受受体主要组织相容性复合体(major histocompatibility complex, MHC)型别的限制,且mRNA理论上可实现不同目的蛋白的体内表达,这使得开发mRNA药物更具灵活性,可用于预防和治疗多种难治性或遗传性疾病。介绍了mRNA的一级结构和高级结构,综述了mRNA药物的临床应用进展,以期帮助理解mRNA的药物功能和临床应用,为mRNA药物的发展提供方向。

关键词: mRNA结构, mRNA药物, 临床应用

Abstract:

Because of the characters of effective, safe and easy to be produced on a large scale, mRNA drugs have showed great potential on preventing viral infections. mRNA represents an emerging field of precision medicine, several mRNA drugs targeted therapies for infectious diseases and cancer have shown beneficial results both in vivo and in vitro. mRNA is stable, immunogenic, and not limited by the receptor's major histocompatibility complex (MHC) type. In theory, mRNA can achieve in vivo expression of different target proteins, which makes the development of mRNA drugs more flexible and can be used to prevent and treat many refractory or hereditary diseases. This paper introduced the primary structure and higher structure of mRNA, and summarized the progress of mRNA drugs in clinical application, which was expected to help understand the drug function of mRNA, and the clinical application, and provide the direction for the development of mRNA drugs.

Key words: mRNA structure, mRNA drugs, clinical application

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