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Gene Therapy for Parkinson's Disease

11 August 2012


Background: Parkinson’s disease (PD) is a neurodegenerative disorder of unknown cause. The characteristic motor impairments of PD including resting tremor, rigidity, slowed movement, decreased dexterity, small handwriting, flexed posture, gait disorder, and imbalance predominantly arise from the loss of neurons in the substantia nigra region of the midbrain that produce the neurotransmitter dopamine. Dopamine replacement therapy provides temporary relief of motor symptoms, but chronic use leads to serious side effects and cannot prevent disease progression. This systematic review will focus upon gene therapy as a possible treatment for PD.

Methods: An exhaustive literature search was conducted in Medline, CINAHL, Web of Science, Google Scholar, and EBMRmultifile, using the search terms gene therapy and Parkinson’s disease in combination and alone as well as terms known to be synonymous. The search was limited to the English language, clinical trials and double-blind, randomized, controlled trials.

Results: Two studies were reviewed based on the inclusion and exclusion criteria delineated in the methods section. Both studies were double-blind, randomized, controlled trials and utilized sham surgery for comparison. Marks et al showed adeno-associated type-2 vector (AAV2)-neurturin delivery in the putamen was not superior to sham surgery. LeWitt et al showed AAV2-glutamic acid decarboxylase (GAD) delivery in the subthalamic nucleus was superior to sham surgery.

Conclusion: This systematic review shows gene therapy may prove to be a treatment option for patients with advanced Parkinson’s disease in the future. More research and development of gene therapy are needed.


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