Novel Gene-Based Therapeutics Targeting the Dopaminergic System in Parkinson's Disease

Deniz Kirik, M. Tomas Björklund, Shilpa Ramaswamy, Jeffrey H. Kordower

Research output: Chapter in Book/Report/Conference proceedingChapter


Novel therapeutic intervention based on gene therapy has moved the field of Parkinson's disease (PD) research forward during the last decade. The process of supplementing cells with genes that promote normal, healthy function promises to be an efficient way of treating diseases like PD, above and beyond what it has been possible to achieve with traditional pharmacotherapy or deep brain stimulation. Studies examining gene therapy for PD usually have one of two goals: (1) to replace dopamine (DA) that is depleted in the striatum or (2) to administer factors that would prevent the degeneration of dopaminergic neurons in the substantia nigra (SN), as this disease is known to lead to a dramatic reduction in levels of DA in the striatum due to the loss and dysfunction of nigral neurons. Several techniques to target the dopaminergic system in the brain have entered into the clinical testing phase using these currently experimental procedures, and others are expected to be tested in the near future. This chapter discusses the status of these therapeutic interventions in both animal models and patients.

Original languageEnglish (US)
Title of host publicationDopamine Handbook
PublisherOxford University Press
ISBN (Electronic)9780199865543
ISBN (Print)9780195373035
StatePublished - Feb 1 2010
Externally publishedYes


  • Dopamine
  • Dopaminergic system
  • Gene therapy
  • Parkinson's disease
  • Substantia nigra
  • Treatment

ASJC Scopus subject areas

  • General Neuroscience


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