RNA与重大疾病诊疗  

RNAs in the diagnosis and treatment of major diseases

作　　者：黄迪[1] 张天英 江小华[3] 黎江 闫章才[4] 孙瑞娟[4] 苏士成[1] 洪微[4] Di Huang;Tianying Zhang;Xiaohua Jiang;Jiang Li;Zhangcai Yan;Ruijuan Sun;Shicheng Su;Wei Hong(Breast Tumor Center,Sun Yat-Sen Memorial Hospital,Sun Yat-Sen University,Guangzhou 510120,China;School of Public Health,Xiamen University,Xiamen 361102,China;The First Affiliated Hospital,University of Science and Technology of China,Hefei 230001,China;Department of Health Sciences,National Natural Science Foundation of China,Beijing 100085,China)

机构地区：[1]中山大学孙逸仙纪念医院乳腺肿瘤中心,广州510120 [2]厦门大学公共卫生学院,厦门361102 [3]中国科学技术大学附属第一医院,合肥230001 [4]国家自然科学基金委员会医学科学部,北京100085

出　　处：《科学通报》2024年第33期4824-4844,共21页Chinese Science Bulletin

摘　　要：RNA参与了机体生命活动的调控,在肿瘤、传染病等重大疾病的发生发展中发挥了重要作用,但由于其种类繁多、结构复杂、功能多样,且易突变和降解,使得RNA作为预防和诊疗靶点在临床中的应用面对诸多挑战.本文围绕RNA生物学机制及其在疾病进程中的调控作用、RNA作为重大疾病诊断的生物标志物及其检测手段、RNA药物和基因编辑在重大疾病治疗中的应用及新策略、传染病及肿瘤RNA疫苗的设计策略及转化临床应用、RNA研究的新技术和新方法等方面,综述了RNA在重大疾病诊疗中的重要发现及前沿,提出RNA在重大疾病诊疗中所面临的挑战,凝练了RNA研究的关键科学问题和未来发展方向.该文指出,利用中国临床资源优势及在RNA功能和机制研究方面的前期创新性发现,加强多学科交叉研究,深入系统地阐释RNA生物学机制及其在疾病中的调控作用,挖掘新的RNA诊疗靶点,开发原创的RNA药物和疫苗,全链条可持续地推动RNA基础研究和临床转化研究,为RNA在重大疾病预防及精准诊疗中的应用提供策略.RNAs(ribonucleic acids),the molecules of“Central Dogma”in life sciences,are comprised of messenger RNAs(mRNAs),ribosomal RNAs(rRNAs),transfer RNAs(tRNAs),small nuclear RNAs(snRNAs),small nucleolar RNAs(snoRNAs),microRNAs(miRNAs),and long non-coding RNAs(lncRNAs),circular RNAs(circRNAs)etc.With the indepth research on RNAs,new types of RNAs with different mechanisms have been identified,which dynamically regulate the physiological processes at multiple levels such as transcription,post-transcriptional regulation and translation.More importantly,RNAs play important roles in the occurrence and development of various major diseases,including malignant tumors,infectious diseases,autoimmune diseases and so on.Theoretical findings of basic RNA research have laid the foundation for the application of RNAs in the diagnosis and treatment of diseases.Therefore,targeting RNA is an emerging strategy in the prevention,diagnosis,and treatment of diseases,which provides novel precision therapy for many previously incurable diseases.However,the RNA diversity,its complex structure,diverse functions,and its susceptibility for mutation and degradation,have brought many challenges to the clinical application.Based on the discussion of the“RNAs in the Diagnosis and Treatment of Major Diseases”at the 357th Shuang Qing Forum of the National Natural Science Foundation of China that was held in Hangzhou on November 20‒22,2023,this review focuses on the regulatory roles and the underlying biological mechanisms of RNAs,the application of RNAs as biomarkers in disease diagnosis,the new strategies and application of RNA-based drugs and gene-editing in the treatment of major disease,the design strategies and translational clinical application of RNA vaccines for infectious diseases and malignant tumors,as well as the new technologies and methods of RNA research.This paper reviews the important discoveries and cutting-edge research of RNAs in the diagnosis and treatment of major diseases,emphasizes the significant challenges for the d

关 键 词：RNA 重大疾病 诊断 治疗 疫苗 新技术 

分 类 号：R440[医药卫生—诊断学]