Asklepios BioPharmaceutical, Inc., Columbus, OH, USA.
Department of Neurology, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
J Parkinsons Dis. 2021;11(s2):S173-S182. doi: 10.3233/JPD-212724.
At present there is a significant unmet need for clinically available treatments for Parkinson's disease (PD) patients to stably restore balance to dopamine network function, leaving patients with inadequate management of symptoms as the disease progresses. Gene therapy is an attractive approach to impart a durable effect on neuronal function through introduction of genetic material to reestablish dopamine levels and/or functionally recover dopaminergic signaling by improving neuronal health. Ongoing clinical gene therapy trials in PD are focused on enzymatic enhancement of dopamine production and/or the restoration of the nigrostriatal pathway to improve dopaminergic network function. In this review, we discuss data from current gene therapy trials for PD and recent advances in study design and surgical approaches.
目前，临床上需要可用于帕金森病 (PD) 患者的治疗方法，以稳定地恢复多巴胺网络功能的平衡，但目前的治疗方法无法满足患者的需求，导致患者在疾病进展过程中无法得到充分的症状管理。基因治疗是一种有吸引力的方法，可以通过引入遗传物质来恢复多巴胺水平和/或通过改善神经元健康来恢复多巴胺能信号传导，从而对神经元功能产生持久的影响。目前正在进行的 PD 临床基因治疗试验集中在酶促增强多巴胺产生和/或恢复黑质纹状体通路，以改善多巴胺能网络功能。在这篇综述中，我们讨论了目前 PD 基因治疗试验的数据以及研究设计和手术方法的最新进展。
