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Helper-dependent adenoviral vectors as gene deliveryvehicles

机译：依赖辅助基因的腺病毒载体作为基因传递载体

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Adenoviral (Ad)-mediated in vivo gene transfer and expression is limited in part by cellular immune responses to viral-encoded proteins. In an attempt to diminish these responses, we have previously developed and described helper- dependent (HD) Ad vectors in which the viral protein coding sequences are completely deleted. These vectors provided efficient delivery, and greater safety which represents a significant advance over existing Ad vectors. In addition, the inherent enhanced insert capacity (up to ~ 37kb) allows for the insertion of large or multiple genes, including expression regulatory sequences. Several drug-regulated gene expression systems are now available for controlling target gene transcription through the use of small-molecule inducing compounds. While early experiments have demonstrated the utility of inducible systems in cell culture and transgenic mice, continued evaluation of such systems in viral gene therapy vectors should lead to discoveries and improvements which will make them amenable for use in a therapeutic context. The generation of a gene therapy approach that combines both safe and efficient vector delivery of one or multiple genes of interest and a small molecule-controlled gene expression system will provide a powerful tool for therapeutic intervention.
机译：腺病毒（Ad）介导的体内基因转移和表达部分受到细胞对病毒编码蛋白的免疫反应的限制。为了减少这些反应，我们先前已经开发和描述了其中完全删除了病毒蛋白编码序列的辅助依赖型（HD）Ad载体。这些载体提供了有效的递送和更高的安全性，这代表了与现有Ad载体相比的重大进步。另外，固有的增强的插入能力（高达〜37kb）允许插入大的或多个基因，包括表达调控序列。现在有几种药物调节的基因表达系统可用于通过使用小分子诱导化合物来控制靶基因的转录。尽管早期实验已经证明了诱导系统在细胞培养和转基因小鼠中的实用性，但是在病毒基因治疗载体中对此类系统的持续评估应导致发现和改进，使其适合用于治疗环境。结合了一个或多个目标基因的安全，高效载体递送和小分子控制的基因表达系统的基因治疗方法的产生，将为治疗干预提供强大的工具。

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