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Mov Disord. 2013 Jan;28(1):96-109. doi: 10.1002/mds.25344.

Trophic factor gene therapy for Parkinson's disease.

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1
Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois 60612, USA. jkordowe@rush.edu

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.

PMID:
23390096
DOI:
10.1002/mds.25344
[Indexed for MEDLINE]
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