Department of Molecular Neuroscience, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.
J Neurol. 2011 Feb;258(2):179-88. doi: 10.1007/s00415-010-5796-9. Epub 2010 Oct 21.
Gene therapy represents a potentially useful additional technique to ameliorate the motor symptoms of Parkinson's disease (PD), and the motor complications of its treatment. The neurodegenerative process itself, as well as the non-motor symptoms of PD, both remain less amenable to most of the current gene therapy approaches. This review presents an overview of the four gene therapies in phase I/II clinical trials, outlines some of the challenges they face, and proposes additional alternative strategies that might improve the clinical prospects of gene therapy for PD. In so doing, we hope to highlight the issue of the current absence of effective treatment for non-motor symptoms of PD and the potential of further candidate targets for gene therapy intervention that might improve upon this, for both specific individuals with genetic forms of PD as well as "sporadic" PD patients.
基因治疗代表了一种潜在有用的附加技术,可以改善帕金森病(PD)的运动症状和其治疗的运动并发症。神经退行性过程本身以及 PD 的非运动症状仍然不太适应大多数当前的基因治疗方法。本综述介绍了正在进行的 I/II 期临床试验中的四种基因治疗方法,概述了它们面临的一些挑战,并提出了可能改善 PD 基因治疗临床前景的其他替代策略。这样做,我们希望强调当前缺乏有效的 PD 非运动症状治疗方法的问题,以及通过基因治疗干预进一步候选靶点的潜力,这可能会改善特定遗传形式的 PD 患者以及“散发性”PD 患者的情况。