Literature DB >> 20631185

Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF.

Adrian P Kells1, Jamie Eberling, Xiaomin Su, Philip Pivirotto, John Bringas, Piotr Hadaczek, Wade C Narrow, William J Bowers, Howard J Federoff, John Forsayeth, Krystof S Bankiewicz.   

Abstract

Clinical studies to date have failed to establish therapeutic benefit of glial cell-derived neurotrophic factor (GDNF) in Parkinson's disease (PD). In contrast to previous nonclinical neuroprotective reports, this study shows clinically relevant and long-lasting regeneration of the dopaminergic system in rhesus macaques lesioned with 1-methy-4-phenyl-1,2,3,6-tetrahydropyridine 3-6 months before GDNF gene delivery (AAV2-GDNF). The observed progressive amelioration of functional deficits, recovery of dopamine, and regrowth of fibers to the striatal neuropil demonstrate that high GDNF expression in the putamen promotes restoration of the dopaminergic system in a primate model of advanced PD. Extensive distribution of GDNF within the putamen and transport to the severely lesioned substantia nigra, after convection-enhanced delivery of AAV2-GDNF into the putamen, indicates anterograde transport via striatonigral connections and is anticipated to occur in PD patients. Overall, these data demonstrate nonclinical neurorestoration after putaminal infusion of AAV2-GDNF and suggest that clinical investigation in PD patients is warranted.

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Year:  2010        PMID： 20631185      PMCID： PMC2914692          DOI： 10.1523/JNEUROSCI.0942-10.2010

Source DB:  PubMed          Journal:  J Neurosci        ISSN： 0270-6474            Impact factor:   6.167


  50 in total

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