Gene Therapy for Parkinson's Disease

Current pharmacological and surgical treatments for Parkinson's disease offer symptomatic improvements to those suffering from this incurable degenerative neurological disorder, but none of these has convincingly shown effects on disease progression. Novel approaches based on gene therapy have...

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Main Authors: Rachel Denyer, Michael R. Douglas
Format: Article
Language:English
Published: Wiley 2012-01-01
Series:Parkinson's Disease
Online Access:http://dx.doi.org/10.1155/2012/757305
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author Rachel Denyer
Michael R. Douglas
author_facet Rachel Denyer
Michael R. Douglas
author_sort Rachel Denyer
collection DOAJ
description Current pharmacological and surgical treatments for Parkinson's disease offer symptomatic improvements to those suffering from this incurable degenerative neurological disorder, but none of these has convincingly shown effects on disease progression. Novel approaches based on gene therapy have several potential advantages over conventional treatment modalities. These could be used to provide more consistent dopamine supplementation, potentially providing superior symptomatic relief with fewer side effects. More radically, gene therapy could be used to correct the imbalances in basal ganglia circuitry associated with the symptoms of Parkinson's disease, or to preserve or restore dopaminergic neurons lost during the disease process itself. The latter neuroprotective approach is the most exciting, as it could theoretically be disease modifying rather than simply symptom alleviating. Gene therapy agents using these approaches are currently making the transition from the laboratory to the bedside. This paper summarises the theoretical approaches to gene therapy for Parkinson's disease and the findings of clinical trials in this rapidly changing field.
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series Parkinson's Disease
spelling doaj-art-25f09cfab34a4b79881b1190400201952025-02-03T05:51:33ZengWileyParkinson's Disease2090-80832042-00802012-01-01201210.1155/2012/757305757305Gene Therapy for Parkinson's DiseaseRachel Denyer0Michael R. Douglas1Department of Neurology, University Hospitals Birmingham NHS Foundation Trust, Mindelson Way, Birmingham B15 2WB, UKSchool of Clinical and Experimental Medicine, University of Birmingham, Edgbaston, Birmingham B15 2TT, UKCurrent pharmacological and surgical treatments for Parkinson's disease offer symptomatic improvements to those suffering from this incurable degenerative neurological disorder, but none of these has convincingly shown effects on disease progression. Novel approaches based on gene therapy have several potential advantages over conventional treatment modalities. These could be used to provide more consistent dopamine supplementation, potentially providing superior symptomatic relief with fewer side effects. More radically, gene therapy could be used to correct the imbalances in basal ganglia circuitry associated with the symptoms of Parkinson's disease, or to preserve or restore dopaminergic neurons lost during the disease process itself. The latter neuroprotective approach is the most exciting, as it could theoretically be disease modifying rather than simply symptom alleviating. Gene therapy agents using these approaches are currently making the transition from the laboratory to the bedside. This paper summarises the theoretical approaches to gene therapy for Parkinson's disease and the findings of clinical trials in this rapidly changing field.http://dx.doi.org/10.1155/2012/757305
spellingShingle Rachel Denyer
Michael R. Douglas
Gene Therapy for Parkinson's Disease
Parkinson's Disease
title Gene Therapy for Parkinson's Disease
title_full Gene Therapy for Parkinson's Disease
title_fullStr Gene Therapy for Parkinson's Disease
title_full_unstemmed Gene Therapy for Parkinson's Disease
title_short Gene Therapy for Parkinson's Disease
title_sort gene therapy for parkinson s disease
url http://dx.doi.org/10.1155/2012/757305
work_keys_str_mv AT racheldenyer genetherapyforparkinsonsdisease
AT michaelrdouglas genetherapyforparkinsonsdisease