Rosewell Amanda, Vetrini Francesco, Ng Philip
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, 77030 USA.
J Genet Syndr Gene Ther. 2011 Oct 29;Suppl 5. doi: 10.4172/2157-7412.s5-001.
Helper-dependent adenoviral vectors are devoid of all viral coding sequences, possess a large cloning capacity, and can efficiently transduce a wide variety of cell types from various species independent of the cell cycle to mediate long-term transgene expression without chronic toxicity. These non-integrating vectors hold tremendous potential for a variety of gene transfer and gene therapy applications. Here, we review the production technologies, applications, obstacles to clinical translation and their potential resolutions, and the future challenges and unanswered questions regarding this promising gene transfer technology.
辅助依赖型腺病毒载体不含所有病毒编码序列，具有大容量克隆能力，能有效转导来自不同物种的多种细胞类型，且不依赖细胞周期，可介导长期转基因表达而无慢性毒性。这些非整合型载体在多种基因转移和基因治疗应用中具有巨大潜力。在此，我们综述了其生产技术、应用、临床转化的障碍及其潜在解决方案，以及关于这项有前景的基因转移技术的未来挑战和未解决的问题。
