• Disease-a-month : DM · Jul 2024

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

    • Chiranjeevee R Saravanan, Reem Faiz Hussein Eisa, Elizabeth Gaviria, Amani Algubari, Kiran Kishor Chandrasekar, Pugazhendi Inban, Priyadarshi Prajjwal, Hyma Bamba, Gurmehar Singh, Mohammed Dheyaa Marsool Marsool, and Srikanth Gadam.
    • Internal Medicine, Madras Medical College, India.
    • Dis Mon. 2024 Jul 1; 70 (7): 101754101754.

    BackgroundParkinson'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.MethodsOnline 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.ResultsGene 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.ConclusionIn comparison to conventional therapies, gene therapy may be advantageous for PD. It may minimize side effects, relieve symptoms, and offer dependable dopamine replacement.Copyright © 2024. Published by Elsevier Inc.

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