Site-specific genomic strategies for gene therapy.
Site-specific genomic strategies for gene therapy.
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发表时间：
2003-08
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通讯作者：
Joylette L. Portlock;M. Calos
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作者：
Joylette L. Portlock;M. Calos
像任何疾病治疗一样，基因治疗应该是安全有效的。安全性可以通过直接纠正缺陷基因本身或通过确保转基因的基因组整合是位点特异性的来解决。不幸的是，已经证明难以在不伴随基因校正或插入效率的损失的情况下实现这种安全水平。在这篇综述中，最近的研究试图实现有效的位点特异性基因治疗，包括使用靶向基因转换，腺相关病毒载体和位点特异性噬菌体重组酶的策略进行了讨论。我们相信这些方法有望实现位点特异性、安全有效的基因治疗。
Like any disease treatment, gene therapy should be safe and efficacious. Safety can be addressed by directly correcting the defective gene itself or by ensuring that genomic integration of a transgene is site-specific. Unfortunately, it has proven difficult to achieve this level of safety without a concomitant loss in efficiency of gene correction or insertion. In this review, recent research attempts to achieve efficient site-specific gene therapy, including strategies using targeted gene conversion, adeno-associated virus vectors and site-specific bacteriophage recombinases are discussed. We believe that these approaches hold promise for site-specific, safe and efficient gene therapy.