Neurodegenerative Studies Laboratory, Brain Mind Institute, Ecole Polytechnique Fédérale de Lausanne (EPFL), CH-1015 Lausanne, Switzerland.
Cold Spring Harb Perspect Med. 2012 Apr;2(4):a009431. doi: 10.1101/cshperspect.a009431.
With the recent development of effective gene delivery systems, gene therapy for the central nervous system is finding novel applications. Here, we review existing viral vectors and discuss gene therapy strategies that have been proposed for Parkinson's disease. To date, most of the clinical trials were based on viral vectors to deliver therapeutic transgenes to neurons within the basal ganglia. Initial trials used genes to relieve the major motor symptoms caused by nigrostriatal degeneration. Although these new genetic approaches still need to prove more effective than existing symptomatic treatments, there is a need for disease-modifying strategies. The investigation of the genetic factors implicated in Parkinson's disease is providing precious insights in disease pathology that, combined with innovative gene delivery systems, will hopefully offer novel opportunities for gene therapy interventions to slow down, or even halt disease progression.
随着有效的基因传递系统的最新发展，中枢神经系统的基因治疗正在找到新的应用。在这里，我们回顾了现有的病毒载体，并讨论了针对帕金森病提出的基因治疗策略。迄今为止，大多数临床试验都是基于病毒载体将治疗性转基因递送到基底神经节中的神经元。最初的试验使用基因来缓解黑质纹状体变性引起的主要运动症状。尽管这些新的遗传方法仍需要证明比现有的对症治疗更有效，但需要有疾病修饰策略。对帕金森病中涉及的遗传因素的研究为疾病病理学提供了宝贵的见解，这些见解与创新的基因传递系统相结合，有望为减缓甚至阻止疾病进展的基因治疗干预提供新的机会。
