Latchman D S
Institute of Child Health, University College London, 30 Guilford Street, WC1N 1EH, London, UK.
Gene. 2001 Feb 7;264(1):1-9. doi: 10.1016/s0378-1119(01)00322-5.
The development of efficient means of delivery genes in vivo is essential both for testing gene function in the intact animal and for human gene therapy procedures. A number of viral and non-viral gene delivery methods have been developed for this purpose. Of those herpes simplex virus (HSV)-based vectors have particular advantages for gene delivery to the nervous system including their ability to infect non-dividing neurones and establish asymptomatic latent infections. Moreover, considerable progress has been made, firstly, in disabling HSV vectors so as to prevent the damaging effects of wild type virus and secondly, to ensure long-term expression of the inserted transgene(s). These vectors thus offer a valuable tool for testing gene function in neuronal cells in vivo and may ultimately be safe enough for use in human gene therapy procedures.
开发高效的体内基因递送方法对于在完整动物中测试基因功能以及人类基因治疗程序而言都至关重要。为此已开发出多种病毒和非病毒基因递送方法。其中,基于单纯疱疹病毒(HSV)的载体在基因递送至神经系统方面具有特殊优势,包括它们能够感染非分裂神经元并建立无症状潜伏感染。此外,已取得了相当大的进展,首先是使HSV载体失活以防止野生型病毒的破坏作用,其次是确保插入的转基因能够长期表达。因此,这些载体为在体内神经元细胞中测试基因功能提供了一种有价值的工具,并且最终可能足够安全以用于人类基因治疗程序。