关键词: Dopaminergic neurons Gene therapy Motor dysfunction Neurodegeneration Parkinson's disease

Mesh : Humans Parkinson Disease / therapy genetics Genetic Therapy / methods Treatment Outcome

来  源:   DOI:10.1016/j.disamonth.2024.101754

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.
CONCLUSIONS: 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,其特征是大脑中多巴胺能神经元的进行性丧失。尽管现有的治疗方法,对于可以阻止或逆转疾病进展的疗法,仍有未满足的需求.基因疗法已经尝试并测试了各种疾病,包括PD.本系统综述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:在线数据库PubMed/Medline,和Cochrane用于筛选本系统综述的研究。使用标准工具评估纳入研究的偏倚风险。
结果:基因疗法可以修复疾病中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回路异常。而不仅仅是治疗症状,这种神经保护方法改变了疾病本身。基因治疗药物目前在患者的床边给药。它可以过度激活与运动功能障碍相关的特定脑回路。PD疗法发展迅速,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。当选择高级疗法时,除了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:与常规疗法相比,基因治疗可能有利于PD。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品。
公众号