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Gene and Cell-Based Therapies for Parkinson’s Disease: Where Are We?
Neurotherapeutics ( IF 5.6 ) Pub Date : 2020-10-30 , DOI: 10.1007/s13311-020-00940-4
Philip C Buttery 1, 2 , Roger A Barker 2, 3
Affiliation  

Parkinson’s disease (PD) is a neurodegenerative disorder that carries large health and socioeconomic burdens. Current therapies for PD are ultimately inadequate, both in terms of symptom control and in modification of disease progression. Deep brain stimulation and infusion therapies are the current mainstay for treatment of motor complications of advanced disease, but these have very significant drawbacks and offer no element of disease modification. In fact, there are currently no agents that are established to modify the course of the disease in clinical use for PD. Gene and cell therapies for PD are now being trialled in the clinic. These treatments are diverse and may have a range of niches in the management of PD. They hold great promise for improved treatment of symptoms as well as possibly slowing progression of the disease in the right patient group. Here, we review the current state of the art for these therapies and look to future strategies in this fast-moving field.



中文翻译:


帕金森病的基因和细胞疗法:我们在哪里?



帕金森病 (PD) 是一种神经退行性疾病,给健康和社会经济带来巨大负担。目前的帕金森病治疗方法无论是在症状控制还是在改变疾病进展方面最终都是不够的。深部脑刺激和输注疗法是目前治疗晚期疾病运动并发症的主要方法,但它们具有非常显着的缺点,并且没有提供疾病改变的元素。事实上,目前还没有任何药物能够在临床上用于改变帕金森病的病程。 PD 的基因和细胞疗法目前正在临床试验。这些治疗方法多种多样,并且可能在帕金森病的治疗中具有一系列的优势。它们对于改善症状治疗以及可能减缓正确患者群体的疾病进展有着巨大的希望。在这里,我们回顾了这些疗法的当前技术水平,并展望了这个快速发展领域的未来策略。

更新日期:2020-11-02
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