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Liver-targeted hydrodynamic gene therapy: Recent advances in the technique

机译：肝靶向水动力基因疗法：该技术的最新进展

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摘要

One of the major research focuses in the field of gene therapy is the development of clinically applicable, safe, and effective gene-delivery methods. Since the first case of human gene therapy was performed in 1990, a number of gene-delivery methods have been developed, evaluated for efficacy and safety, and modified for human application. To date, viral-vector-mediated deliveries have shown effective therapeutic results. However, the risk of lethal immune response and carcinogenesis have been reported, and it is still controversial to be applied as a standard therapeutic option. On the other hand, delivery methods for nonviral vector systems have been developed, extensively studied, and utilized in in vivo gene-transfer studies. Compared to viral-vector mediated gene transfer, nonviral systems have less risk of biological reactions. However, the lower gene-transfer efficiency was a critical hurdle for applying them to human gene therapy. Among a number of nonviral vector systems, our studies focus on hydrodynamic gene delivery to utilize physical force to deliver naked DNA into the cells in the living animals. This method achieves a high gene-transfer level by DNA solution injections into the tail vein of rodents, especially in the liver. With the development of genome editing methods, in vivo gene-transfer therapy using this method is currently the focus in this research field. This review explains the method principle, efficiency, safety, and procedural modifications to achieve a high level of reproducibility in large-animal models.
机译：基因治疗领域的主要研究之一是开发临床适用，安全和有效的基因递送方法。自从1990年进行人类基因治疗的第一例以来，​​已经开发了许多基因传递方法，对其有效性和安全性进行了评估，并对人类应用进行了修改。迄今为止，病毒载体介导的递送已显示出有效的治疗结果。然而，已经报道了致死性免疫反应和致癌作用的风险，将其用作标准治疗选择仍存在争议。另一方面，已经开发，广泛研究了非病毒载体系统的递送方法，并将其用于体内基因转移研究。与病毒载体介导的基因转移相比，非病毒系统的生物反应风险较小。然而，较低的基因转移效率是将其应用于人类基因治疗的关键障碍。在许多非病毒载体系统中，我们的研究集中在流体动力学基因传递上，以利用物理力将裸露的DNA传递到活体动物的细胞中。通过将DNA溶液注射到啮齿动物的尾静脉中，尤其是在肝脏中，该方法达到了很高的基因转移水平。随着基因组编辑方法的发展，使用这种方法的体内基因转移治疗目前是该研究领域的重点。这篇综述解释了方法原理，效率，安全性和程序修改，以在大型动物模型中实现高水平的可重复性。

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