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

IF 3.8 3区 医学 Q1 MEDICINE, GENERAL & INTERNAL Dm Disease-A-Month Pub Date : 2024-06-06 DOI:10.1016/j.disamonth.2024.101754
Chiranjeevee R. Saravanan MBBS , Reem Faiz Hussein Eisa MBBS , Elizabeth Gaviria MD , Amani Algubari MD , Kiran Kishor Chandrasekar MBBS , Pugazhendi Inban MD , Priyadarshi Prajjwal MBBS , Hyma Bamba MBBS , Gurmehar Singh MBBS , Mohammed Dheyaa Marsool Marsool MBChB , Srikanth Gadam MD
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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.

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帕金森病基因治疗方法的有效性和安全性:系统综述。
背景:帕金森病(PD)是一种神经退行性疾病,其特征是大脑中多巴胺能神经元的逐渐丧失。尽管已有一些治疗方法,但对于能阻止或逆转疾病进展的疗法的需求仍未得到满足。基因疗法已在包括帕金森病在内的多种疾病中进行了尝试和测试。本系统综述旨在评估基因治疗技术在帕金森病临床试验中的安全性和有效性:本系统综述使用在线数据库 PubMed/Medline 和 Cochrane 筛选研究。采用标准工具评估了纳入研究的偏倚风险:基因疗法可以修复因疾病而受损的多巴胺能神经元,或治疗与帕金森病症状相关的基底节回路异常。这种神经保护方法不仅能治疗症状,还能改变疾病本身。基因治疗的药物目前是在病人床边使用的。它可以过度激活与运动功能障碍相关的特定脑回路。帕金森病疗法发展迅速,但目前还没有足够的正面试验来评估现有疗法的安全性和有效性。在选择先进疗法时,除了考虑每种治疗方案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,基因疗法可能对帕金森病具有优势。结论:与传统疗法相比,基因疗法可能对帕金森病有优势,它可以最大限度地减少副作用,缓解症状,并提供可靠的多巴胺替代。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Dm Disease-A-Month
Dm Disease-A-Month 医学-医学:内科
CiteScore
5.70
自引率
2.50%
发文量
140
审稿时长
>12 weeks
期刊介绍: Designed for primary care physicians, each issue of Disease-a-Month presents an in-depth review of a single topic. In this way, the publication can cover all aspects of the topic - pathophysiology, clinical features of the disease or condition, diagnostic techniques, therapeutic approaches, and prognosis.
期刊最新文献
Table of Contents Information for Readers Title Page C2: Editorial Board Table of Contents
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