Viral vectors for gene delivery to the central nervous system.
Viral vectors for gene delivery to the central nervous system.
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DOI：
10.1016/j.nbd.2011.09.014
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发表时间：
2012-11
影响因子：
6.1
通讯作者：
Samulski, R. Jude
中科院分区：
文献类型：
--
作者：
Lentz, Thomas B.;Gray, Steven J.;Samulski, R. Jude
关键词：
基因治疗对神经系统疾病如帕金森氏症、肌萎缩侧索硬化症（ALS）、癫痫和阿尔茨海默氏症的潜在益处是巨大的。即使严重症状的出现有所延迟，对患有这些疾病和其他疾病的患者也是非常宝贵的。已经在开发能够将治疗性基因递送至CNS以治疗神经病症的载体方面进行了大量努力。在潜在载体的最前沿，病毒系统已经进化到可以有效地将其遗传物质传递到细胞中。不同病毒的生物学为基因治疗的挑战提供了独特的解决方案，例如细胞靶向，转基因表达和载体生产。重要的是要考虑载体的天然生物学，当决定它是否将是最有效的特定治疗功能。在这篇综述中，我们概述了理想的载体基因传递到中枢神经系统的功能，并讨论如何以及可用的病毒载体相比，这种模式。包括腺相关病毒、逆转录病毒、腺病毒和疱疹病毒载体。重点放在自然生物学的功能，使这些病毒的有效工具，基因传递，重点是它们在中枢神经系统中的应用。我们的目标是提供洞察最佳载体的功能，以及哪些病毒载体可以提供这些功能。
The potential benefits of gene therapy for neurological diseases such as Parkinson’s, Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer’s are enormous. Even a delay in the onset of severe symptoms would be invaluable to patients suffering from these and other diseases. Significant effort has been placed in developing vectors capable of delivering therapeutic genes to the CNS in order to treat neurological disorders. At the forefront of potential vectors, viral systems have evolved to efficiently deliver their genetic material to a cell. The biology of different viruses offers unique solutions to the challenges of gene therapy, such as cell targeting, transgene expression and vector production. It is important to consider the natural biology of a vector when deciding whether it will be the most effective for a specific therapeutic function. In this review, we outline desired features of the ideal vector for gene delivery to the CNS and discuss how well available viral vectors compare to this model. Adeno-associated virus, retrovirus, adenovirus and herpesvirus vectors are covered. Focus is placed on features of the natural biology that have made these viruses effective tools for gene delivery with emphasis on their application in the CNS. Our goal is to provide insight into features of the optimal vector and which viral vectors can provide these features.
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影响因子：
5.4
作者：
Akache, Bassel;Grimm, Dirk;Kay, Mark A.
通讯作者：
Kay, Mark A.
DOI：
10.1073/pnas.86.8.2525
发表时间：
1989-04-01
影响因子：
11.1
作者：
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通讯作者：
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影响因子：
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作者：
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通讯作者：
Castro, Maria G.
影响因子：
5.4
作者：
Amalfitano, A;Hauser, MA;Chamberlain, JS
通讯作者：
Chamberlain, JS