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Development of advanced therapies based on viral vector-mediated overexpression of therapeutic molecules and knockdown of disease-related genes for Parkinson's disease

机译:基于病毒载体介导的治疗分子过表达和疾病相关基因敲除的帕金森氏病先进疗法的发展

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摘要

The last decade witnessed the translation of several gene-based therapeutic approaches from experimental studies to early clinical trials. Studies targeting the treatment of Parkinson's disease (PD) were among the forefront of trials in the CNS. In this article, we overview three major strategies for the treatment of PD: the enzyme-replacement strategies are based on well-defined principles of functional restoration and are well suited for treatment of patients with advanced disease who would typically experience complications due to side effects of pharmacotherapy. Neurotrophic factor delivery, on the other hand, aims to delay the disability and eventually modifiy disease progression. Finally, we present an outlook to a completely new way of interfering with the disease process, which is taking advantage of recently discovered RNAi mechanisms in cells. Gene therapy is now becoming a reality in the clinics and developments in the next decade will help uncover the true potential of this approach for not only the treatment of PD patients, but also many other neurological disorders.
机译:过去十年见证了从实验研究到早期临床试验的几种基于基因的治疗方法的转化。针对帕金森氏病(PD)的治疗研究是中枢神经系统研究的前沿领域。在本文中,我们概述了治疗PD的三种主要策略:酶替代策略基于功能恢复的明确定义原则,非常适合治疗因副作用而通常会出现并发症的晚期疾病患者药物治疗。另一方面,神经营养因子的递送旨在延迟残疾并最终改变疾病的进展。最后,我们提出了一种全新的干扰疾病过程的方法,该方法利用了细胞中最近发现的RNAi机制。基因治疗现在正在临床中成为现实,在未来十年中,其发展将不仅有助于发现这种方法真正的潜力,不仅可以治疗PD患者,还可以治疗许多其他神经系统疾病。

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