Gene Therapy for Parkinson's Disease
Gene Therapy for Parkinson's Disease
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DOI：
10.1002/mds.22785
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发表时间：
2010-01-01
影响因子：
8.6
通讯作者：
Kordower, Jeffrey H.
中科院分区：
文献类型：
--
作者：
Bjorklund, Tomas;Kordower, Jeffrey H.
帕金森病 (PD) 患者将接受基因治疗，以试图减轻症状并可能改变其病程，这一曾经梦幻般的理论概念现已成为现实。基于积极的临床前数据，四种不同的基因治疗方法目前正在进行 I 期或 II 期临床试验。一些方法旨在增加内源性多巴胺的水平或增强前药左旋多巴的功能。其他药物旨在通过减少特定大脑结构（例如丘脑底核）与 PD 相关的过度活动来使基底神经节回路正常化。每一种都是为了缓解症状。最后，营养因子的基因传递不仅可以增强多巴胺能功能，而且有可能改善疾病，拥有强大的临床前数据库，并且也正在进行临床试验。本综述中讨论了每种方法。 (C) 2010 运动障碍学会
The once fantastic theoretical concept that patients with Parkinson's disease (PD) would receive gene therapy in an attempt to alleviate their symptoms and potentially modify the course of their disease has become a reality. On the basis of positive preclinical data, four different gene therapy approaches are currently in Phase I or Phase II clinical trials. Some approaches are intended to increase levels of endogenous dopamine or enhance the function of the prodrug levodopa. Others are intended to normalize basal ganglia circuitry by reducing the PD-related overactivity of specific brain structures such as the subthalamic nucleus. Each is intended for symptomatic benefit. Finally, gene delivery of trophic factors that not only augment dopaminergic function but are potentially disease modifying has a strong preclinical database and are also in clinical trials. Each of these approaches is discussed in the present review. (C) 2010 Movement Disorder Society