Dopaminergic neurons protected from degeneration by GDNF gene therapy

Derek L. Choi-Lundberg, Qing Lin, Yung Nien Chang, Yawen L. Chiang, Carl M. Hay, Hasan Mohajeri, Beverly L. Davidson, Martha C. Bohn*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

577 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)838-841
Number of pages4
Issue number5301
StatePublished - Feb 7 1997

ASJC Scopus subject areas

  • General


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