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帕金森病的基因治疗:神经退行性疾病的最新治疗方法。

Gene therapy for Parkinson's disease: state-of-the-art treatments for neurodegenerative disease.

机构信息

School of Clinical and Experimental Medicine, University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK.

出版信息

Expert Rev Neurother. 2013 Jun;13(6):695-705. doi: 10.1586/ern.13.58.

Abstract

Pharmacological and surgical treatments offer symptomatic benefits to patients with Parkinson's disease; however, as the condition progresses, patients experience gradual worsening in symptom control, with the development of a range of disabling complications. In addition, none of the currently available therapies have convincingly shown disease-modifying effects - either in slowing or reversing the disease. These problems have led to extensive research into the possible use of gene therapy as a treatment for Parkinson's disease. Several treatments have reached human clinical trial stages, providing important information on the risks and benefits of this novel therapeutic approach, and the tantalizing promise of improved control of this currently incurable neurodegenerative disorder.

摘要

药物治疗和手术治疗为帕金森病患者提供了症状缓解的益处;然而,随着病情的发展,患者会逐渐出现症状控制的恶化,并出现一系列致残性并发症。此外,目前可用的治疗方法均未显示出具有明显的疾病改善作用——无论是减缓还是逆转疾病。这些问题促使人们广泛研究基因治疗作为帕金森病的一种治疗方法的可能性。一些治疗方法已经进入人体临床试验阶段,为这种新的治疗方法的风险和益处提供了重要信息,并为这种目前无法治愈的神经退行性疾病的更好控制带来了诱人的前景。

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