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基因治疗方法在帕金森病中的疗效和安全性:系统评价。

The efficacy and safety of gene therapy approaches in Parkinson's disease: A systematic review.

机构信息

Internal Medicine, Madras Medical College, India.

Ain Shams University, Cairo, Egypt.

出版信息

Dis Mon. 2024 Jul;70(7):101754. doi: 10.1016/j.disamonth.2024.101754. Epub 2024 Jun 6.

Abstract

BACKGROUND

Parkinson's disease (PD) is a neurodegenerative disorder characterized by the progressive loss of dopaminergic neurons in the brain. Despite existing treatments, there remains an unmet need for therapies that can halt or reverse disease progression. Gene therapy has been tried and tested for a variety of illnesses, including PD. The goal of this systematic review is to assess gene therapy techniques' safety and effectiveness in PD clinical trials.

METHODS

Online databases PubMed/Medline, and Cochrane were used to screen the studies for this systematic review. The risk of bias of the included studies was assessed using standard tools.

RESULTS

Gene therapy can repair damaged dopaminergic neurons from the illness or deal with circuit anomalies in the basal ganglia connected to Parkinson's disease symptoms. Rather than only treating symptoms, this neuroprotective approach alters the illness itself. Medication for gene therapy is currently administered at the patient's bedside. It can hyperactivate specific brain circuits associated with motor dysfunction. PD therapies are developing quickly, and there aren't enough head-to-head trials evaluating the safety and effectiveness of available treatments. When choosing an advanced therapy, patient-specific factors should be considered in addition to the effectiveness and safety of each treatment option.

CONCLUSION

In comparison to conventional therapies, gene therapy may be advantageous for PD. It may minimize side effects, relieve symptoms, and offer dependable dopamine replacement.

摘要

背景

帕金森病(PD)是一种神经退行性疾病,其特征是大脑中的多巴胺能神经元进行性丧失。尽管存在现有治疗方法,但仍需要能够阻止或逆转疾病进展的治疗方法。基因治疗已被尝试用于治疗多种疾病,包括 PD。本系统评价的目的是评估基因治疗技术在 PD 临床试验中的安全性和有效性。

方法

使用在线数据库 PubMed/Medline 和 Cochrane 筛选本系统评价的研究。使用标准工具评估纳入研究的偏倚风险。

结果

基因治疗可以修复因疾病而受损的多巴胺能神经元,或处理与帕金森病症状相关的基底神经节中的回路异常。这种神经保护方法不仅可以治疗症状,还可以改变疾病本身。基因治疗药物目前在患者床边给药。它可以过度激活与运动功能障碍相关的特定大脑回路。PD 治疗方法正在迅速发展,并且没有足够的头对头试验来评估现有治疗方法的安全性和有效性。在选择先进的治疗方法时,除了每种治疗方法的有效性和安全性外,还应考虑患者的具体因素。

结论

与传统疗法相比,基因治疗可能对 PD 具有优势。它可能会最小化副作用、缓解症状并提供可靠的多巴胺替代。

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