Trophic Factor Gene Therapy for Parkinson's Disease

Jeffrey H. Kordower, Anders Bjorklund

Research output: Contribution to journalReview articlepeer-review

100 Scopus citations

Abstract

Parkinson's disease (PD) is a chronic and progressive neurodegenerative movement disorder for which there is presently no cure. Pharmacological remedies targeting the dopaminergic network are relatively effective at ameliorating motor deficits, especially in the early stages of the disease, but none of these therapies are curative and many generate their own problems. Recent advances in PD research have demonstrated that gene delivery of trophic factors, glial cell line-derived neurotrophic factor (GDNF) and neurturin, in particular, can provide structural and functional recovery in rodent and nonhuman primate models of PD. Similar success has been gleaned in open-label clinical trials, although this has yet to be realized in double-blinded analyses. This work reviews the field of trophic factor gene delivery for PD.

Original languageEnglish (US)
Pages (from-to)96-109
Number of pages14
JournalMovement Disorders
Volume28
Issue number1
DOIs
StatePublished - Jan 2013
Externally publishedYes

Keywords

  • GDNF
  • Neuroprotection
  • Neurturin
  • Parkinson's disease
  • Regeneration

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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