Gene therapy for Parkinson's disease

Tomas Bjorklund, Jeffrey H. Kordower

Research output: Contribution to journalArticlepeer-review

44 Scopus citations


The once fantastic theoretical concept that patients with Parkinson's disease (PD) would receive gene therapy in an attempt to alleviate their symptoms and potentially modify the course of their disease has become a reality. On the basis of positive preclinical data, four different gene therapy approaches are currently in Phase I or Phase II clinical trials. Some approaches are intended to increase levels of endogenous dopamine or enhance the function of the prodrug levodopa. Others are intended to normalize basal ganglia circuitry by reducing the PD-related overactivity of specific brain structures such as the subthalamic nucleus. Each is intended for symptomatic benefit. Finally, gene delivery of trophic factors that not only augment dopaminergic function but are potentially disease modifying has a strong preclinical database and are also in clinical trials. Each of these approaches is discussed in the present review.

Original languageEnglish (US)
Pages (from-to)S161-S173
JournalMovement Disorders
Issue numberSUPPL. 1
StatePublished - 2010
Externally publishedYes


  • Clinical trials
  • Dopamine
  • Gene therapy
  • Nonhuman primates
  • Trophic factors

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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