An imposing obstacle to gene therapy is the inability to transduce all of the necessary cells in a target organ. This certainly applies to gene transfer to the brain, especially when one considers the challenges involved in scaling up transduction from animal models to use in the clinic. Non-neurotropic viral gene transfer vectors (e.g., adenovirus, adeno-associated virus, and lentivirus) do not spread very far in the nervous system, and consequently these vectors transduce brain regions mostly near the injection site in adult animals. This indicates that numerous, well-spaced injections would be required to achieve widespread transduction in a large brain with these vectors. In contrast, herpes simplex virus type 1 (HSV-1) is a promising vector for widespread gene transfer to the brain owing to the innate ability of the virus to spread through the nervous system and form latent infections in neurons that last for the lifetime of the infected individual. In this review, we summarize the published literature of the transduction patterns produced by attenuated HSV-1 vectors in small animals as a function of the injection site, and discuss the implications of the distribution for widespread gene transfer to the large animal brain.

译文

:基因治疗的一个潜在障碍是无法在靶器官中转导所有必需的细胞。这当然适用于基因向大脑的转移,尤其是当考虑到将动物模型的转导规模扩大到临床使用时所面临的挑战时。非神经营养性病毒基因转移载体(例如,腺病毒,腺伴随病毒和慢病毒)在神经系统中不会传播很远,因此,这些载体主要在成年动物的注射部位附近转导脑区域。这表明,使用这些载体在大脑中实现广泛的转导将需要大量间隔良好的注射。相比之下,单纯疱疹病毒1型(HSV-1)是一种有前途的载体,可以广泛地将基因转移到大脑,原因是该病毒具有先天的能力,可以在神经系统中传播并在神经元中形成潜伏的感染,这种感染可以持续一生。受感染的个人。在这篇综述中,我们总结了已减毒的HSV-1载体在小动物中产生的转导模式作为注射部位的函数的已发表文献,并讨论了该分布对于将基因广泛转移到大动物脑中的意义。

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