Advances of adeno-associated virus applied in gene therapy to hemophilia from bench work to the clinical use

查看全文 作　　者：Xiaolei [1,4]Pei;Mingzhe [4]Han;Lei [1,2,3]Zhang

机构地区：[1]State Key Laboratory of Experimental Hematology,Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Tianjin,China;[2]Tianjin Key Laboratory of Gene Therapy for Blood Diseases,Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Tianjin,China;[3]CAMS Key Laboratory of Gene Therapy for Blood Diseases,Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Tianjin,China;[4]Hematopoietic Stem Cell Transplantation Center,Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Tianjin,China

出　　处：《Blood Science》索引2019年第1卷第2期,共7页

基　　金：supported by the Beijing-Tianjin-Hebei basic research project 18JCZDJC44600/H2018206423 Lei Zhang;Non-profit Central Research Institute Fund of Chinese Academy of Medical Sciences 2017PT31047,2018PT31038/2018PT32028;CAMS Innovation Fund for Medical Sciences(CIFMS)2016-I2M-1-018;Novartis Foundation. 摘　　要：Hemophilia A and B are diseases caused by a single gene deficiency and are thus suitable for gene therapy.In recent clinical research,adeno-associated virus(AAV)was employed by several teams in the treatment of hemophilia A and B,and the outcomes were encouraging.In this review,we summarized the most recent research on the mechanism and application of AAV in the treatment of hemophilia,trying to analyze the advantages of AAV gene therapy and the main challenges in its clinical use.We also summarized the clinical trials involving hemophilia,especially those employing AAV gene therapy to treat hemophilia A and B,some of which have already been completed and some that are still ongoing.From the reports of the completed clinical trials,we tried to determine the correlations among AAV dose,AAV serotype,immune response,and gene expression time.Finally,taking into account the most recent studies investigating AAV capsid modification,transgene optimization,and AAV chaperones,we summarized the direction of basic research and clinical applications of AAV in the future. 关键词：Adeno-associated virus HEMOPHILIA FVIII FIX Clinical trial

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Advances of adeno-associated virus applied in gene therapy to hemophilia from bench work to the clinical use

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