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帕金森病基因治疗的当前实验研究

Current Experimental Studies of Gene Therapy in Parkinson's Disease.

作者信息

Lin Jing-Ya, Xie Cheng-Long, Zhang Su-Fang, Yuan Weien, Liu Zhen-Guo

机构信息

Department of Neurology, Xinhua Hospital Affiliated to the Medical School of Shanghai JiaoTong UniversityShanghai, China.

Department of Neurology, The first Affiliated Hospital of Wenzhou Medical University, Wenzhou Medical UniversityWenzhou, China.

出版信息

Front Aging Neurosci. 2017 May 3;9:126. doi: 10.3389/fnagi.2017.00126. eCollection 2017.

Abstract

Parkinson's disease (PD) was characterized by late-onset, progressive dopamine neuron loss and movement disorders. The progresses of PD affected the neural function and integrity. To date, most researches had largely addressed the dopamine replacement therapies, but the appearance of L-dopa-induced dyskinesia hampered the use of the drug. And the mechanism of PD is so complicated that it's hard to solve the problem by just add drugs. Researchers began to focus on the genetic underpinnings of Parkinson's disease, searching for new method that may affect the neurodegeneration processes in it. In this paper, we reviewed current delivery methods used in gene therapies for PD, we also summarized the primary target of the gene therapy in the treatment of PD, such like neurotrophic factor (for regeneration), the synthesis of neurotransmitter (for prolong the duration of L-dopa), and the potential proteins that might be a target to modulate via gene therapy. Finally, we discussed RNA interference therapies used in Parkinson's disease, it might act as a new class of drug. We mainly focus on the efficiency and tooling features of different gene therapies in the treatment of PD.

摘要

帕金森病(PD)的特征为发病较晚、多巴胺神经元进行性丧失以及运动障碍。帕金森病的进展影响神经功能和完整性。迄今为止,大多数研究主要关注多巴胺替代疗法,但左旋多巴诱发的运动障碍的出现阻碍了该药物的使用。而且帕金森病的机制非常复杂,仅靠添加药物很难解决问题。研究人员开始关注帕金森病的遗传基础,寻找可能影响其中神经退行性变过程的新方法。在本文中,我们综述了目前用于帕金森病基因治疗的递送方法,我们还总结了基因治疗在帕金森病治疗中的主要靶点,如神经营养因子(用于再生)、神经递质的合成(用于延长左旋多巴的作用时间),以及可能通过基因治疗进行调节的潜在蛋白质。最后,我们讨论了用于帕金森病的RNA干扰疗法,它可能成为一类新型药物。我们主要关注不同基因疗法在帕金森病治疗中的效率和工具特性。

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Current Experimental Studies of Gene Therapy in Parkinson's Disease.帕金森病基因治疗的当前实验研究
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