Palmer Donna J, Ng Philip
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Methods Mol Biol. 2008;433:33-53. doi: 10.1007/978-1-59745-237-3_3.
Helper-dependent adenoviral vectors (HDAd) are deleted of all viral coding sequences and have demonstrated tremendous potential for gene therapy by providing long-term, high-level transgene expression in the absence of chronic toxicity. Thus, HDAd are superior to early generation Ad for gene therapy of genetic diseases where long-term transgene expression is required. This chapter describes in detail the rescue, amplification, and large-scale production of HDAd.
辅助依赖型腺病毒载体(HDAd)删除了所有病毒编码序列,通过在无慢性毒性的情况下提供长期、高水平的转基因表达,已显示出在基因治疗方面的巨大潜力。因此,对于需要长期转基因表达的遗传性疾病的基因治疗,HDAd优于早期一代腺病毒。本章详细描述了HDAd的拯救、扩增和大规模生产。