Gene therapy clinical trials worldwide to 2017: An update

被引:599
作者
Ginn, Samantha L. [1 ,2 ]
Amaya, Anais K. [1 ,2 ]
Alexander, Ian E. [1 ,2 ,3 ]
Edelstein, Michael [4 ]
Abedi, Mohammad R. [5 ]
机构
[1] Univ Sydney, Childrens Med Res Inst, Gene Therapy Res Unit, Westmead, NSW, Australia
[2] Sydney Childrens Hosp Network, Westmead, NSW, Australia
[3] Univ Sydney, Discipline Child & Adolescent Hlth, Westmead, NSW, Australia
[4] Ctr Global Hlth Secur, Chatham House, London, England
[5] Uppsala Univ Hosp, Dept Lab Med, Uppsala, Sweden
关键词
clinical trials; database; gene therapy; The Journal of Gene Medicine; worldwide; DUCHENNE MUSCULAR-DYSTROPHY; T-CELLS; LENTIVIRAL VECTOR; DOUBLE-BLIND; BONE-MARROW; STEM-CELLS; OPEN-LABEL; GENOME; RNA; SAFETY;
D O I
10.1002/jgm.3015
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our November 2017 update, we have entries on 2597 trials undertaken in 38 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and the genes that have been transferred. Details of the analyses presented, and our searchable database are available via The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: . We also provide an overview of the progress being made in gene therapy clinical trials around the world, and discuss key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies, which have the potential to transform the field moving forward.
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页数:16
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