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Dopaminergic neurons protected from degeneration by GDNF gene therapy
journal contributionposted on 2023-05-16, 15:31 authored by Derek Choi-LundbergDerek Choi-Lundberg, Lin, Q, Chang, YN, Chiang, YL, Hay, CM, Mohajeri, H, Davidson, BL, Bohn, MC
Glial cell line-derived neurotrophic factor (GDNF) supports growth and survival of dopaminergic (DA) neurons. A replication-defective adenoviral (Ad) vector encoding human GDNF injected near the rat substantia nigra was found to protect DA neurons from the progressive degeneration induced by the neurotoxin 6- hydroxydopamine (6-OHDA) injected into the striatum. Ad GDNF gene therapy reduced loss of DA neurons approximately threefold 6 weeks after 6-OHDA lesion, as compared with no treatment or injection of Ad lacZ or Ad mGDNF (encoding a biologically inactive deletion mutant GDNF). These results suggest that Ad vector-mediated GDNF gene therapy may slow the DA neuronal cell loss in humans with Parkinson's disease.
Department/SchoolTasmanian School of Medicine
PublisherAmerican Association for the Advancement of Science
Place of publicationWashington