Trophic factor gene therapy for Parkinson's disease

JH Kordower, A Bjorklund - Movement Disorders, 2013 - Wiley Online Library
JH Kordower, A Bjorklund
Movement Disorders, 2013Wiley Online Library
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 …
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. © 2013 Movement Disorder Society
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