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使用基于单纯疱疹病毒载体的基因治疗。

Gene therapy using herpes simplex virus-based vectors.

作者信息

Latchman D S

机构信息

Institute of Child Health, University College London, UK.

出版信息

Histol Histopathol. 2000 Oct;15(4):1253-9. doi: 10.14670/HH-15.1253.

Abstract

Gene therapy involves the use of specific genes to treat human diseases and is thus critically dependent on efficient gene delivery systems. Although a variety of systems for such gene delivery are under development, HSV has unique advantages in terms of its large genome size and for gene delivery in the nervous system because of its ability to enter a latent state in neuronal cells. Considerable progress has been made in the effective disablement of this virus whilst retaining its ability to deliver genes and in producing long term expression of the foreign gene. Although much remains to be achieved in the further disablement of the virus and its testing in rodent and primate models of human diseases, it is likely that these viruses may ultimately be of use in human gene therapy procedures particularly for otherwise intractable neurological diseases.

摘要

基因治疗涉及使用特定基因来治疗人类疾病,因此严重依赖于高效的基因递送系统。尽管目前正在开发多种用于此类基因递送的系统,但单纯疱疹病毒(HSV)因其基因组较大,且能够在神经元细胞中进入潜伏状态,在神经系统的基因递送方面具有独特优势。在有效使这种病毒失活的同时保留其递送基因的能力以及实现外源基因的长期表达方面已经取得了相当大的进展。尽管在进一步使病毒失活并在人类疾病的啮齿动物和灵长类动物模型中进行测试方面仍有许多工作要做,但这些病毒最终可能会用于人类基因治疗程序,特别是针对其他难以治疗的神经疾病。

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