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首页> 外文期刊>Expert opinion on biological therapy >Gene therapy for Parkinson's disease using recombinant adeno-associated viral vectors.
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Gene therapy for Parkinson's disease using recombinant adeno-associated viral vectors.

机译：使用重组腺相关病毒载体对帕金森氏病进行基因治疗。

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Existing strategies for gene therapy in the treatment of Parkinson's disease include the delivery of genes encoding dopamine (DA)-synthesising enzymes, leading to localised production of DA in the striatum; genes encoding factors that protect nigral neurons against ongoing degeneration, such as glial cell line-derived neurotrophic factor; and genes encoding proteins that produce the inhibitory transmitter gamma-aminobutylic acid (GABA) in the subthalamic nucleus (STN), thus suppressing the hyperactive STN. Recombinant adeno-associated viral (rAAV) vectors, which are derived from non-pathogenic viruses, have been shown to be suitable for clinical trials. These rAAVs have been found to transduce substantial numbers of neurons efficiently and to express transgenes in mammalian brains for long periods of time, with minimum inflammatory and immunological responses. In vivo imaging using positron emission tomography is useful for monitoring transgene expression and for assessing the functional effects of gene delivery. Vector systems that regulate transgene expression are necessary to increase safety in clinical applications, and the development of such systems is in progress.
机译：用于治疗帕金森氏病的基因治疗的现有策略包括传递编码多巴胺（DA）合成酶的基因，从而导致纹状体中DA的局部产生。编码保护黑素神经元免于持续退化的因子的基因，例如神经胶质细胞源性神经营养因子;以及编码在丘脑下核（STN）中产生抑制性递质γ-氨基丁酸（GABA）的蛋白质的基因，从而抑制了过度活跃的STN。衍生自非病原性病毒的重组腺相关病毒（rAAV）载体已被证明适用于临床试验。已经发现这些rAAV有效地转导大量神经元，并在哺乳动物脑中长时间表达转基因，具有最小的炎症和免疫反应。使用正电子发射断层扫描的体内成像可用于监测转基因表达和评估基因传递的功能效果。调节转基因表达的载体系统对于提高临床应用的安全性是必需的，并且这种系统的开发正在进行中。

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