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基因修饰体细胞国内外临床研究和相关产业现状及未来发展趋势
引用本文:王征旭,游嘉,武立华,童春容,吴迪,刘彩霞,昌晓红,程洪艳,程金莲,王少华,曹彩,马洁. 基因修饰体细胞国内外临床研究和相关产业现状及未来发展趋势[J]. 药物评价研究, 2021, 0(2): 250-264
作者姓名:王征旭  游嘉  武立华  童春容  吴迪  刘彩霞  昌晓红  程洪艳  程金莲  王少华  曹彩  马洁
作者单位:中国人民解放军总医院第七医学中心;高博医学(血液病)北京研究中心北京博仁医院;北京医院生物治疗中心;北京大学人民医院;首都医科大学附属北京中医医院;中关村玖泰药物临床试验技术创新联盟
基金项目:国家科技重大专项课题——重大新药创制(2016ZX09101094);北京市科委企业技术创新平台建设(Z181100000518028)。
摘    要:基因编辑技术可针对性增强体细胞的靶向性,一定程度上解决了传统过继性免疫疗法在肿瘤等多种疾病治疗中显露出的靶向性差等问题.最具代表性的是嵌合抗原受体T细胞(CAR-T)免疫疗法,其在血液系统肿瘤治疗中已取得良好疗效,此外,T细胞受体基因工程化的T细胞(TCR-T)、基因修饰的树突状细胞以及基因修饰的干细胞在多种疾病治疗的...

关 键 词:嵌合抗原受体T细胞(CAR-T)  自然杀伤(NK)细胞  CAR-NK  T细胞受体基因工程化的T细胞(TCR-T)  基因修饰树突状细胞  基因修饰干细胞

Current situation and future development on clinical research and related industries of genetically modified somatic cells
WANG Zhengxu,YOU Jia,WU Lihua,TONG Chunrong,WU Di,LIU Caixia,CHANG Xiaohong,CHENG Hongyan,CHENG Jinlian,WANG Shaohua,CAO Cai,MA Jie. Current situation and future development on clinical research and related industries of genetically modified somatic cells[J]. Drug Evaluation Research, 2021, 0(2): 250-264
Authors:WANG Zhengxu  YOU Jia  WU Lihua  TONG Chunrong  WU Di  LIU Caixia  CHANG Xiaohong  CHENG Hongyan  CHENG Jinlian  WANG Shaohua  CAO Cai  MA Jie
Affiliation:(Seventh Medical Center of Chinese PLA General Hospital,Beijing 100700,China;Beijing Boren Hospital,GoBroad Medical Institute of Hematology,GoBroad Healthcare Group,Beijing 100070,China;Center of Biotherapy,Beijing Hospital,National Center of Gerontology,Institute of Geriatric Medicine,Chinese Academy of Medical Sciences,Beijing 100730,China;Peking University People's Hospital,Beijing 100044,China;Beijing Hospital of Traditional Chinese Medicine,Capital Medical University,Beijing 100010,China;Zhongguancun Jiutai Good Clinical Practice Union,Beijing 100101,China)
Abstract:The Gene editing technology could enhance the on-target of somatic cells,and partially resolve the problem of off-target of the adoptive immunotherapy in the treatment of tumors and the other diseases.One of the he most representative methods is CAR-T cell therapy,which has been achieved well curative effect in the treatment of hematological tumors.In addition,the treatment methods which include using TCR-T cells,genetically modified dendritic cells and genetically modified stem cells have also been shown well safety and effectiveness in the clinical research of various diseases.In view of this,many research institutions and biological companies in China and the other countries have accelerated their deployment in the fields of genetically modified somatic cell at now,and will be reaching a period of rapid development.The clinical transformation and application of genetically modified somatic cell therapy hold great promising in the fulture.
Keywords:chimeric antigen receptors-modified T cells(CAR-T)  natural killer(NK)cell  CAR-NK  T cell receptor(TCR)-T cell  gene-modified dendritic cell(DC)  gene-modified stem cells
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