{"title":"The efficacy and safety of gene therapy approaches in Parkinson's disease: A systematic review","authors":"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","doi":"10.1016/j.disamonth.2024.101754","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><p>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.</p></div><div><h3>Methods</h3><p>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.</p></div><div><h3>Results</h3><p>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.</p></div><div><h3>Conclusion</h3><p>In comparison to conventional therapies, gene therapy may be advantageous for PD. It may minimize side effects, relieve symptoms, and offer dependable dopamine replacement.</p></div>","PeriodicalId":51017,"journal":{"name":"Dm Disease-A-Month","volume":"70 7","pages":"Article 101754"},"PeriodicalIF":3.8000,"publicationDate":"2024-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Dm Disease-A-Month","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0011502924000804","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
引用次数: 0
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.
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.