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

doi:10.1126/science.275.5301.838

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

Pediatrics

Research output: Contribution to journal › Article › peer-review

582 Scopus citations

Abstract

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 language	English (US)
Pages (from-to)	838-841
Number of pages	4
Journal	Science
Volume	275
Issue number	5301
DOIs	https://doi.org/10.1126/science.275.5301.838
State	Published - Feb 7 1997

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title = "Dopaminergic neurons protected from degeneration by GDNF gene therapy",

abstract = "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.",

author = "Choi-Lundberg, {Derek L.} and Qing Lin and Chang, {Yung Nien} and Chiang, {Yawen L.} and Hay, {Carl M.} and Hasan Mohajeri and Davidson, {Beverly L.} and Bohn, {Martha C.}",

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T1 - Dopaminergic neurons protected from degeneration by GDNF gene therapy

AU - Choi-Lundberg, Derek L.

AU - Lin, Qing

AU - Chang, Yung Nien

AU - Chiang, Yawen L.

AU - Hay, Carl M.

AU - Mohajeri, Hasan

AU - Davidson, Beverly L.

AU - Bohn, Martha C.

PY - 1997/2/7

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N2 - 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.

AB - 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.

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Dopaminergic neurons protected from degeneration by GDNF gene therapy

Abstract

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