Knockdown of GAD67 protein levels normalizes neuronal activity in a rat model of Parkinson's disease

Journal of Gene Medicine

Volumn 13, Issue 3, 2011, Pages 188-197

  Horvath, Lazlo ^a   van Marion, Ingrid ^a   Taï, Khalid ^a   Nielsen, Troels Tolstrup ^b,c   Lundberg, Cecilia ^a  

^a LUND UNIVERSITY   (Sweden)

^b UNIVERSITY OF COPENHAGEN   (Denmark)

^c Rigshospitalet   (Denmark)

Author keywords

6 hydroxydopamine lesion; GAD67; Lentiviral vector; MtCO1; Parkinson's disease; RNAi; Striatum; Synthetic miRNA

Indexed keywords

BRAIN PROTEIN; DOPAMINE; GLUTAMATE DECARBOXYLASE 67; LENTIVIRUS VECTOR; MICRORNA; MITOCHONDRIAL RNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CATECHOLAMINE DEPLETION; CONTROLLED STUDY; CORPUS STRIATUM; FEMALE; NERVE CONDUCTION; NONHUMAN; NUCLEOTIDE SEQUENCE; PARKINSON DISEASE; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN EXPRESSION; RAT; RNA INTERFERENCE; SUBSTANTIA NIGRA; WESTERN BLOTTING;

ANIMALS; BLOTTING, WESTERN; CELL LINE; DISEASE MODELS, ANIMAL; DOPAMINE; ELECTRON TRANSPORT COMPLEX IV; FEMALE; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GENE KNOCKDOWN TECHNIQUES; GENETIC VECTORS; GLUTAMATE DECARBOXYLASE; GREEN FLUORESCENT PROTEINS; HUMANS; IMMUNOHISTOCHEMISTRY; LENTIVIRUS; MICRORNAS; MITOCHONDRIA; NEOSTRIATUM; PARKINSON DISEASE; RATS; RATS, SPRAGUE-DAWLEY; RNA INTERFERENCE; RNA, SMALL INTERFERING; TIME FACTORS;

PARKINSONIA; RATTUS;

EID: 79955129749     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1555     Document Type: Article

References (35)

1. Lewitt PA, Rezai AR, Leehey MA, et al. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol 2011; 10(4): 309-319.
2. Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 2007; 369: 2097-2105.
3. Carta AR, Fenu S, Pala P, et al. Selective modifications in GAD67 mRNA levels in striatonigral and striatopallidal pathways correlate to dopamine agonist priming in 6-hydroxydopamine-lesioned rats. Eur J Neurosci 2003; 18: 2563-2572.
4. Bergman H, Deuschl G. Pathophysiology of Parkinson's disease: from clinical neurology to basic neuroscience and back. Mov Disord 2002; 3: S28-S40.
5. Winkler C, Bentlage C, Cenci MA, Nikkhah G, Björklund A. Regulation of neuropeptide mRNA expression in the basal ganglia by intrastriatal and intranigral transplants in the rat Parkinson model. Neuroscience 2003; 118: 1063-1077.
6. Wichmann T, DeLong MR. Pathophysiology of Parkinson's disease: the MPTP primate model of the human disorder. Ann NY Acad Sci 2003; 991: 199-213.
7. Soghomonian JJ, Gonzales C, Chesselet MF. Messenger RNAs encoding glutamate-decarboxylases are differentially affected by nigrostriatal lesions in subpopulations of striatal neurons. Brain Res 1992; 576: 68-79.
8. Dostrovsky JO, Hutchison WD, Lozano AM. The globus pallidus, deep brain stimulation, and Parkinson's disease. Neuroscientist 2002; 8: 284-290.
9. Goetz CG, Poewe W, Rascol O, Sampaio C. Evidence-based medical review update: pharmacological and surgical treatments of Parkinson's disease: 2001-2004. Mov Disord 2005; 20: 523-539.
10. Lundberg C, Björklund T, Carlsson T, et al. Applications of lentiviral vectors for biology and gene therapy of neurological disorders. Curr Gene Ther 2008; 8: 461-473.
11. Nielsen TT, Marion I, Hasholt L, Lundberg C. Neuron-specific RNA interference using lentiviral vectors. J Gene Med 2009; 11: 559-569.
12. Van den Haute C, Eggermont K, Nuttin B, Debyser Z, Baekelandt V. Lentiviral vector-mediated delivery of short hairpin RNA results in persistent knockdown of gene expression in mouse brain. Hum Gene Ther 2003; 14: 1799-1807.
13. Stegmeier F, Hu G, Rickles RJ, Hannon GJ, Elledge SJ. A lentiviral microRNA-based system for single-copy polymerase II-regulated RNA interference in mammalian cells. Proc Natl Acad Sci USA 2005; 102: 13212-13217.
14. Wiznerowicz M, Trono D. Conditional suppression of cellular genes: lentivirus vector-mediated drug-inducible RNA interference. J Virol 2003; 77: 8957-8961.
15. Nielsen TT, Jakobsson J, Rosenqvist N, Lundberg C. Incorporating double copies of a chromatin insulator into lentiviral vectors results in less viral integrants. BMC Biotechnol 2009; 9: 13.
16. Sachdeva R, Jönsson ME, Nelander J, et al. Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors. Proc Natl Acad Sci USA 2010; 107: 11602-7.
17. Ericson C, Wictorin K, Lundberg C. Ex vivo and in vitro studies of transgene expression in rat astrocytes transduced with lentiviral vectors. Exp Neurol 2002; 173: 22-30.
18. Georgievska B, Kirik D, Rosenblad C, Lundberg C, Björklund A. Neuroprotection in the rat Parkinson model by intrastriatal GDNF gene transfer using a lentiviral vector. Neuroreport 2002; 13: 75-82.
19. McBride JL, Boudreau RL, Harper SQ, et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci USA 2008; 105: 5868-5873.
20. Boudreau RL, Martins I, Davidson BL. Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther 2009; 7: 169-175.
21. Kirik D, Rosenblad C, Björklund A. Characterization of behavioral and neurodegenerative changes following partial lesions of the nigrostriatal dopamine system induced by intrastriatal 6-hydroxydopamine in the rat. Exp Neurol. 1998; 152: 259-277.
22. Vila M, Levy R, Herrero MT, et al. Consequences of nigrostriatal denervation on the functioning of the basal ganglia in human and nonhuman primates: an in situ hybridization study of cytochrome oxidase subunit I mRNA. J Neurosci 1997; 17: 765-773.
23. Azdad K, Chàvez M, Don Bischop P, et al. Homeostatic plasticity of striatal neurons intrinsic excitability following dopamine depletion. PLoS One 2009; 4: e6908.
24. Vila M, Levy R, Herrero MT, et al. Metabolic activity of the basal ganglia in parkinsonian syndromes in human and non-human primates: a cytochrome oxidase histochemistry study. Neuroscience 1996; 71: 903-912.
25. Levy R, Herrero MT, Ruberg M, et al. Effects of nigrostriatal denervation and L-dopa therapy on the GABAergic neurons in the striatum in MPTP-treated monkeys and Parkinson's disease: an in situ hybridization study of GAD67 mRNA. Eur J Neurosci 1995; 7: 1199-1209
26. Hajeri PB, Singh SK. siRNAs: their potential as therapeutic agents-Part I. Designing of siRNAs. Drug Discov Today 2009; 14: 851-858.
27. Chen Q, Lee TH, Wetsel WC, et al. Reversal of cocaine sensitization-induced behavioral sensitization normalizes GAD67 and GABAA receptor alpha2 subunit expression, and PKC zeta activity. Biochem Biophys Res Commun 2007; 356: 733-8. Epub 2007 Mar 15.
28. Björklund A, Dunnett SB, Brundin P, et al. Neural transplantation for the treatment of Parkinson's disease. Lancet Neurol 2003; 2: 437-445.
29. Abbas-Terki T, Blanco-Bose W, Déglon N, Pralong W, Aebischer P. Lentiviral-mediated RNA interference. Hum Gene Ther 2002; 13: 2197-2201.
30. Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006; 441: 537-541.
31. Giering JC, Grimm D, Storm TA, Kay MA. Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther 2008; 16: 1630-1636.
32. Ulusoy A, Sahin G, Björklund T, Aebischer P, Kirik D. Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal projection neurons. Mol Ther 2009; 17: 1574-1584.
33. Drouet V, Perrin V, Hassig R, et al. Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol 2009; 65: 276-285.
34. Dhar SS, Wong-Riley MT. Coupling of energy metabolism and synaptic transmission at the transcriptional level: role of nuclear respiratory factor 1 in regulating both cytochrome c oxidase and NMDA glutamate receptor subunit genes. J Neurosci 2009; 29: 483-492.
35. Lacombe E, Khaindrava V, Melon C, Oueslati A, Kerkerian-Le Goff L, Salin P Different functional basal ganglia subcircuits associated with anti-akinetic and dyskinesiogenic effects of antiparkinsonian therapies. Neurobiol Dis 2009; 36: 116-125.