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首页> 外文期刊>Human gene therapy. Clinical development >A Hybrid Adenoviral Vector System Achieves Efficient Long-Term Gene Expression in the Liver via piggyBac Transposition
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A Hybrid Adenoviral Vector System Achieves Efficient Long-Term Gene Expression in the Liver via piggyBac Transposition

机译：混合腺病毒载体系统通过piggyBac转座在肝脏中实现高效的长期基因表达

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摘要

Much research has gone into the development of hybrid gene delivery systems that combine the broad tropism and efficient transduction of adenoviral vectors with the ability to achieve stable expression of cargo genes. In addition to gene therapy applications, such a system has considerable advantages for studies of gene function in vivo, permitting fine-tuned genetic manipulation with higher throughput than can be achieved using standard transgenic and DNA targeting techniques. Existing strategies are limited, however, by low integration efficiencies, small cargo capacity, and/or a dependence on target cell division. The utility of this approach could be enhanced by a system that provides all of the following: (1) efficient delivery, (2) stable expression in a high percentage of target cells (whether mitotic or not), (3) large cargo capacity, (4) flexibility to use with a wide range of additional experimental conditions, and (5) simple experimental technique. Here we report the initial characterization of a hybrid system that meets these criteria by utilizing piggyBac (PB) transposition to achieve genomic integration from adenoviral vectors. We demonstrate stable expression of an adenovirus (Ad)-PB-delivered reporter gene in approx 20-40% of hepatocytes following standard tail vein injection. Its high efficiency and flexibility relative to existing hybrid adenoviral gene delivery approaches indicate a considerable potential utility of the Ad-PB system for therapeutic gene delivery and in vivo studies of gene function.
机译：混合基因传递系统的开发已经进行了很多研究，该系统结合了广泛的向性和腺病毒载体的有效转导，并具有实现货物基因稳定表达的能力。除了基因治疗的应用外，这种系统在体内基因功能研究方面也具有相当大的优势，与使用标准转基因和DNA靶向技术相比，该系统可进行具有更高通量的精细遗传操作。然而，现有策略受到集成效率低，货物容量小和/或对靶细胞分裂的依赖性的限制。可以通过提供以下所有功能的系统来增强此方法的实用性：（1）有效递送，（2）在高百分比的靶细胞（无论是否有丝分裂）中稳定表达，（3）大货物容量， （4）在广泛的其他实验条件下使用的灵活性，以及​​（5）简单的实验技术。在这里，我们报告通过利用piggyBac（PB）转座来实现腺病毒载体的基因组整合，满足这些标准的混合系统的初始表征。我们证明在标准尾静脉注射后约20-40％的肝细胞中稳定表达腺病毒（Ad）-PB传递的报告基因。与现有的杂交腺病毒基因传递方法相比，它的高效性和灵活性表明Ad-PB系统在治疗性基因传递和基因功能的体内研究中具有巨大的潜在用途。

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