Current Neurology and Neuroscience Reports

, Volume 11, Issue 4, pp 362–370

An Update on Gene Therapy in Parkinson’s Disease



Gene therapy for Parkinson’s disease (PD) may offer an alternative to current pharmacologic and surgical treatments; the former are limited by motor complications and non-motor adverse effects, and both by lack of neuroprotection. Three main strategies under investigation using gene transfer for targeted protein expression include improving availability of dopamine to the striatum with more continuous delivery, reducing activity in the subthalamic nucleus by locally inducing γ-aminobutyric acid expression, or protecting and restoring nigrostriatal neuronal function with trophic factor expression. This review summarizes the components of gene therapy for PD, the preclinical rationale for each strategy, data from the most recently published clinical trials using four different vector-gene agents, and challenges in moving gene therapy forward. Thus far, safety data from phase 1 trials have been encouraging for all four agents and one phase 2 trial suggests modest symptomatic efficacy, but definitive conclusions on efficacy cannot yet be drawn.


Parkinson’s disease Gene therapy Gene transfer Investigational therapies Viral vectors Surgical treatment Aromatic L-amino acid decarboxylase (AADC) Tyrosine hydroxylase (TH) Guanosine 5’-triphosphate cyclohydrolase 1 (CH1) Subthalamic nucleus Neurturin (NTN) Neurotrophic factors Continuous dopamine delivery Neuroprotection 


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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© Springer Science+Business Media, LLC (outside the USA) 2011

Authors and Affiliations

  1. 1.Department of NeurologyUniversity of California, San FranciscoSan FranciscoUSA
  2. 2.Parkinson’s Disease Research, Education, and Clinical CenterSan Francisco Veterans Affairs Medical CenterSan FranciscoUSA

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