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首页> 外文期刊>Expert opinion on biological therapy >Targeted approaches for gene therapy and the emergence of engineered meganucleases.
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Targeted approaches for gene therapy and the emergence of engineered meganucleases.

机译：基因治疗的靶向方法和工程化大范围核酸酶的出现。

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BACKGROUND: In spite of significant advances in gene transfer strategies in the field of gene therapy, there is a strong emphasis on the development of alternative methods, providing better control of transgene expression and insertion patterns. OBJECTIVE: Several new approaches consist of targeting a desired transgene or gene modification in a well defined locus, and we collectively refer to them as 'targeted approaches'. The use of redesigned meganucleases is one of these emerging technologies. Here we try to define the potential of this method, in the larger scope of targeted strategies. METHODS: We survey the different types of targeted strategies, presenting the achievements and the potential applications, with a special emphasis on the use of redesigned endonucleases. CONCLUSION: redesigned endonucleases represent one of the most promising tools for targeted approaches, and the opening of a clinical trial for AIDS patients has recently shown the maturity of these strategies. However, there is still a 'quest' for the best reagents, that is the endonucleases providing the best efficacy:toxicity ratio. New advances in protein design have allowed the engineering of new scaffolds, such as meganucleases, and the landscape of existing methods is likely to change over the next few years.
机译：背景：尽管基因治疗领域的基因转移策略取得了重大进展，但仍非常重视替代方法的开发，以提供对转基因表达和插入模式的更好控制。目的：几种新方法包括在明确定义的基因座中靶向所需的转基因或基因修饰，我们将它们统称为“靶向方法”。重新设计的大范围核酸酶的使用是这些新兴技术之一。在这里，我们尝试在更大范围的针对性策略中定义这种方法的潜力。方法：我们调查了不同类型的靶向策略，介绍了取得的成就和潜在的应用，特别强调了重新设计的核酸内切酶的使用。结论：重新设计的核酸内切酶代表了靶向治疗方法中最有前途的工具之一，最近针对艾滋病患者开展的临床试验表明，这些策略已经成熟。然而，仍然存在对“最佳试剂”的“追求”，即提供最佳功效：毒性比的核酸内切酶。蛋白质设计的新进展已使新支架的工程设计成为可能，例如大范围核酸酶，并且未来几年现有方法的前景可能会发生变化。

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