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首页> 外文期刊>Current drug targets. CNS and neurological disorders >Liposomal and viral vectors for gene therapy of the central nervous system.
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Liposomal and viral vectors for gene therapy of the central nervous system.

机译：用于中枢神经系统基因治疗的脂质体和病毒载体。

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摘要

Due to the presence of the blood-brain barrier, the central nervous system (CNS) is not easily accessible to systemically delivered macromolecules with therapeutic activity such as growth factors, cytokines or enzymes. Therefore, the expression of exogenously administered genes in the brain has been proposed for a wide variety of inherited and acquired diseases of the CNS, for which classical pharmacotherapy is unavailable or not easily applicable. Gene therapy to the CNS has been the target of a great number of studies aiming at finding a viable therapeutic strategy for the treatment of neurological disorders. This approach has already been used as a promising tool for brain protection and repair from neuronal insults and degeneration in several animal models, and is currently being applied in clinical trials. The choice of an appropriate vector system for transferring the desired gene into the affected brain area is an important issue for developing a safe and efficient gene therapy approach for the CNS. In this review, we focus on the various types of vectors that have been used for gene delivery into the CNS. Particular emphasis is given to their mode of preparation, biological activity, safety and in vivo behavior. Examples illustrating the potential of both viral and non-viral vectors in therapeutic applications to brain disorders are provided. In addition, the use of lentiviral vectors for in vivo modeling of genetic disorders of the CNS is discussed.
机译：由于存在血脑屏障，中枢神经系统（CNS）不容易被具有治疗活性的系统性递送大分子（例如生长因子，细胞因子或酶）访问。因此，已经提出了在脑中外源施用基因的表达用于中枢神经系统的多种遗传性和后天性疾病，对于这些疾病，经典药物疗法不可用或不容易应用。针对中枢神经系统的基因治疗已经成为许多研究的目标，旨在找到可行的神经疾病治疗策略。该方法已被用作在多种动物模型中保护和修复神经元损伤和变性的有前途的工具，目前正用于临床试验。选择合适的载体系统以将期望的基因转移到患病的大脑区域是开发用于CNS的安全有效基因治疗方法的重要问题。在这篇综述中，我们集中于已用于基因传递到中枢神经系统的各种类型的载体。特别强调它们的制备方式，生物学活性，安全性和体内行为。提供了说明病毒和非病毒载体在脑疾病的治疗应用中的潜力的实例。另外，讨论了慢病毒载体在体内建模中枢神经系统遗传疾病中的用途。

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