Gene therapy on the move.
Gene therapy on the move.
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DOI：
10.1002/emmm.201202287
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发表时间：
2013-11
影响因子：
11.1
通讯作者：
Grez, Manuel
中科院分区：
文献类型：
--
作者：
Kaufmann, Kerstin B.;Buening, Hildegard;Galy, Anne;Schambach, Axel;Grez, Manuel
第一个基因治疗临床试验是在二十多年前开始的。在早期，基因治疗与许多实验性医学方法一样，由于在少数接受治疗的患者中发生严重的副作用而受到阻碍。对导致治疗和/或载体相关挫折的分子和细胞机制的理解导致了具有改进的安全性和治疗功效的高度复杂的基因转移工具的开发。利用这些先进的工具，在过去几年中开始了一系列I/II期试验，临床效果良好，迄今没有副作用报告。此外，高效的基因打靶策略和定点基因编辑技术也已开发并应用于临床。到目前为止，已有1900多项临床试验，基因治疗已经从一个愿景走向临床现实。本文综述了基因治疗在遗传性疾病矫正中的应用，早期临床试验中遇到的局限性和缺点，以及基因治疗作为矫正单基因疾病的一种强有力的治疗选择的复兴。
The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the occurrence of severe side effects in a few treated patients. The understanding of the molecular and cellular mechanisms leading to treatment- and/or vector-associated setbacks has resulted in the development of highly sophisticated gene transfer tools with improved safety and therapeutic efficacy. Employing these advanced tools, a series of Phase I/II trials were started in the past few years with excellent clinical results and no side effects reported so far. Moreover, highly efficient gene targeting strategies and site-directed gene editing technologies have been developed and applied clinically. With more than 1900 clinical trials to date, gene therapy has moved from a vision to clinical reality. This review focuses on the application of gene therapy for the correction of inherited diseases, the limitations and drawbacks encountered in some of the early clinical trials and the revival of gene therapy as a powerful treatment option for the correction of monogenic disorders.
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影响因子：
48
作者：
Anliker, Brigitte;Abel, Tobias;Buchholz, Christian J.
通讯作者：
Buchholz, Christian J.
影响因子：
11.1
作者：
Buening, Hildegard
通讯作者：
Buening, Hildegard
影响因子：
5.1
作者：
Brendel, C.;Mueller-Kuller, U.;Grez, M.
通讯作者：
Grez, M.
影响因子：
5.1
作者：
Barde, I.;Laurenti, E.;Trono, D.
通讯作者：
Trono, D.
影响因子：
--
作者：
Brendel, Christian;Haenseler, Walther;Siler, Ulrich
通讯作者：
Siler, Ulrich