Gene therapy for Parkinson's disease

Takao Yasuhara, Isao Date

Research output: Chapter in Book/Report/Conference proceedingChapter

5 Citations (Scopus)


Parkinson's disease is characterized by the degeneration of the nigrostriatal dopaminergic neurons with the manifestation of tremor, rigidity, akinesia, and disturbances of postural reflexes. Medication using L-DOPA and surgeries including deep brain stimulation are the established therapies for Parkinson's disease. Cell therapies are also effective and have rapidly developed with the recent advancement in molecular biological technology including gene transfer. In this review, ex vivo gene therapy using genetically engineered cell transplantation for Parkinson's disease model of animals is described, including catecholamine/neurotrophic factor-secreting cell transplantation with or without encapsulation, as well as in vivo gene therapy using direct injection of viral vector to increase dopamine-production, ameliorate the survival of dopaminergic neurons, correct the deteriorated microenvironment, or normalize genetic abnormality. Furthermore, the future directions for clinical application are described together with recent clinical trials of gene therapy.

Original languageEnglish
Title of host publicationBirth, Life and Death of Dopaminergic Neurons in the Substantia Nigra
PublisherSpringer-Verlag Wien
Number of pages9
ISBN (Print)9783211926598
Publication statusPublished - 2009

Publication series

NameJournal of Neural Transmission, Supplementa
ISSN (Print)0303-6995


  • Cell therapy
  • Dopaminergic neurons
  • Encapsulation
  • Neural stem cell
  • Neurotrophic factor
  • Viral vector

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology
  • Psychiatry and Mental health
  • Biological Psychiatry


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