mRNA药物在遗传性疾病治疗领域的研究进展  

Progress in research on mRNA drugs for the treatment of genetic diseases

作　　者：史文君 程先硕[2] 罗培 马继龙 董坚[2] Shi Wenjun;Cheng Xianshuo;Luo Pei;Ma Jilong;Dong Jian(Department of Oncology,the First People′s Hospital of Yunnan Province,the Affiliated Hospital of Kunming University of Science and Technology,Kunming,Yunnan 650032,China;Department of Colorectal Surgery,the Third Affiliated Hospital of Kunming Medical University,Tumor Hospital of Yunnan Province,Kunming,Yunnan 650118,China)

机构地区：[1]云南省第一人民医院/昆明理工大学附属医院肿瘤内科,昆明650032 [2]昆明医科大学第三附属医院/云南省肿瘤医院结直肠外科,昆明650118

出　　处：《中华医学遗传学杂志》2024年第3期368-376,共9页Chinese Journal of Medical Genetics

基　　金：昆明理工大学医学联合专项(KUST-KH2022031Y)。

摘　　要：mRNA药物近年来在治疗遗传性疾病方面显示出巨大的潜力,吸引了众多研究者的关注。综述回顾过去10年mRNA药物在遗传性疾病治疗领域的研究进展,探讨其作用机制和结构设计,并重点探讨了mRNA药物在替代疗法中所表现出的众多优势,如高特异性、低给药剂量、持续表达等,同时针对mRNA药物有效递送、储存方法等难题展开讨论,以期为后续的研究提供指引。In recent years,mRNA drugs have shown a great potential for the treatment of genetic diseases and attracted the attention of many researchers.This article has reviewed the advance in the research of mRNA drugs for the treatment of genetic diseases over the past 30 years,including their mechanisms of action and structure design,with a focus on their advantages as alternative therapies such as high specificity,low dosage,and sustained expression.Meanwhile,challenges for the effective delivery and storage methods for the mRNA drugs are discussed,with an aim to provide guidance for subsequent researches.

关 键 词：mRNA药物 遗传性疾病 体外转录 

分 类 号：R596[医药卫生—内科学]