Dopaminergic neurons protected from degeneration by GDNF gene therapy

被引:535
作者
ChoiLundberg, DL
Lin, Q
Chang, YN
Chiang, YL
Hay, CM
Mohajeri, H
Davidson, BL
Bohn, MC
机构
[1] UNIV ROCHESTER,DEPT NEUROBIOL & ANAT,ROCHESTER,NY 14642
[2] GENET THERAPY,GAITHERSBURG,MD 20878
[3] UNIV IOWA,COLL MED,DEPT INTERNAL MED,IOWA CITY,IA 52242
关键词
D O I
10.1126/science.275.5301.838
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
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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页码:838 / 841
页数:4
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