Local gene knockdown in the brain using viral-mediated RNA interference

Nature Medicine

Volumn 9, Issue 12, 2003, Pages 1539-1544

  Hommel, Jonathan D ^a   Sears, Robert M ^a   Georgescu, Dan ^a   Simmons, Diana L ^a   DiLeone, Ralph J ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DOPAMINE; PSYCHOSTIMULANT AGENT; TYROSINE 3 MONOOXYGENASE;

ARTICLE; BEHAVIOR; DOPAMINE METABOLISM; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENE FUNCTION; GENE THERAPY; GENETIC MANIPULATION; HUMAN; HUMAN CELL; KNOCKOUT GENE; MESENCEPHALON; MOTOR DYSFUNCTION; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA INTERFERENCE; VIRUS;

ANIMALS; BASE SEQUENCE; BEHAVIOR, ANIMAL; BRAIN; DEPENDOVIRUS; DNA; GENE EXPRESSION; GENE TARGETING; GENETIC VECTORS; HUMANS; MALE; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; RNA INTERFERENCE; TYROSINE 3-MONOOXYGENASE;

EID: 0346216853     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm964     Document Type: Article

References (36)

1. Lewandoski, M. Conditional control of gene expression in the mouse. Nat. Rev. Genet. 2, 743-755 (2001).
2. Gossen, M. & Bujard, H. Studying gene function in eukaryotes by conditional gene inactivation. Annu. Rev. Genet. 36, 153-173 (2002).
3. DiLeone, R.J., Russell, L.B. & Kingsley, D.M. An extensive 3′ regulatory region controls expression of Bmp5 in specific anatomical structures of the mouse embryo. Genetics 148, 401-408 (1998).
4. Fire, A. et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391, 806-811 (1998).
5. Fraser, A.G. et al. Functional genomic analysis of C. elegans chromosome I by systematic RNA interference, Nature 408, 325-330 (2000).
6. Lum, L. et al. Identification of Hedgehog pathway components by RNAi in Drosophila cultured cells. Science 299, 2039-2045 (2003).
7. Kennerdell, J.R. & Carthew, R.W. Heritable gene silencing in Drosophila using double-stranded RNA. Nat. Biotechnol. 18, 896-898 (2000).
8. Kalidas, S. & Smith, D.P. Novel genomic cDNA hybrids produce effective RNA interference in adult Drosophila. Neuron 33, 177-184 (2002).
9. Elbashir, S.M. et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411, 494-498 (2001).
10. Yu, J.Y., DeRuiter, S.L. & Turner, D.L. RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells. Proc. Natl. Acad. Sci. USA 99, 6047-6052 (2002).
11. Krichevsky, A.M. & Kosik, K.S. RNAi functions in cultured mammalian neurons. Proc. Natl. Acad. Sci. USA 99, 11926-11929 (2002).
12. McCaffrey, A.P. et al. RNA interference in adult mice. Nature 418, 38-39 (2002).
13. Xia, H., Mao, Q., Paulson, H.L. & Davidson, B.L. siRNA-mediated gene silencing in vitro and in vivo. Nat. Biotechnol. 20, 1006-1010 (2002).
14. McCown, T.J., Xiao, X., Li, J., Breese, G.R. & Samulski, R.J. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713, 99-107 (1996).
15. Chamberlin, N.L., Du, B., de Lacalle, S. & Saper, C.B. Recombinant adeno-associated virus vector: use for transgene expression and anterograde tract tracing in the CNS. Brain Res. 793, 169-175 (1998).
16. McGeer, E.G., McGeer, P.L. & Wada, J.A. Distribution of tyrosine hydroxylase in human and animal brain. J. Neurochem. 18, 1647-1658 (1971).
17. Han, V.K., Snouweart, J., Towle, A.C., Lund, P.K. & Lauder, J.M. Cellular localization of tyrosine hydroxylase mRNA and its regulation in the rat adrenal medulla and brain by in situ hybridization with an oligodeoxyribonucleotide probe. J. Neurosci. Res. 17, 11-18 (1987).
18. Koob, G.F. & Nestler, E.J. The neurobiology of drug addiction. J. Neuropsychiatry Clin. Neurosci. 9, 482-497 (1997).
19. Berridge, K.C. & Robinson, T.E. What is the role of dopamine in reward: hedonic impact, reward learning, or incentive salience? Brain Res. Brain Res. Rev. 28, 309-369 (1998).
20. Zhou, Q.Y. & Palmiter, R.D. Dopamine-deficient mice are severely hypoactive, adipsic, and aphagic. Cell 83, 1197-1209 (1995).
21. Gibb, W.R. Functional neuropathology in Parkinson's disease. Eur. Neurol. 38, (suppl. 2), 21-50 (1997).
22. Chuang, D., Zsilla, G. & Costa, E. Turnover rate of tyrosine hydroxylase during trans-synaptic induction. Mol. Pharmacol. 11, 784-794 (1975).
23. Moore, K.E. The actions of amphetamine on neurotransmitters: a brief review. Biol. Psychiatry 12, 451-462 (1977).
24. Colotla, V.A., Flores, E., Oscos, A., Meneses, A. & Tapia, R. Effects of MPTP on locomotor activity in mice. Neurotoxicol. Teratol. 12, 405-407 (1990).
25. Rozas, G., Lopez-Martin, E., Guerra, M.J. & Labandeira-Garcia, J.L. The overall rod performance test in the MPTP-treated-mouse model of Parkinsonism. J. Neurosci. Methods 83, 165-175 (1998).
26. Tiscornia, G., Singer, 0., Ikawa, M. & Verma, I.M. A general method for gene knockdown in mice by using lentiviral vectors expressing small interfering RNA. Proc. Natl. Acad. Sci. USA 100, 1844-1848 (2003).
27. Fitzsimons, H.L., McKenzie, J.M. & During, M.J. Insulators coupled to a minimal bidirectional tet cassette for tight regulation of rAAV-mediated gene transfer in the mammalian brain. Gene Ther. 8, 1675-1681 (2001).
28. Yu, J.Y., Taylor, J., DeRuiter, S.L., Vojtek, A.B. & Turner, D.L. Simultaneous inhibition of GSK3α and GSK3β using hairpin siRNA expression vectors. Mol. Ther. 7, 228-236 (2003).
29. Girod, A. et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat. Med. 5, 1052-1056 (1999).
30. Zhou, Q.Y., Quaife, C.J. & Palmiter, R.D. Targeted disruption of the tyrosine hydroxylase gene reveals that catecholamines are required for mouse fetal development, Nature 374, 640-643 (1995).
31. Polymeropoulos, M.H. et al. Mutation in the α-synuclein gene identified in families with Parkinson's disease. Science 276, 2045-2047 (1997).
32. Kitada, T. et al. Mutations in the parkin gene cause autosomal recessive juvenile parkinsonism. Nature 392, 605-608 (1998).
33. Manno, C.S. et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972 (2003)
34. Zolotukhin, S. et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6, 973-985 (1999).
35. Georgescu, D. et al. Involvement of the lateral hypothalamic peptide orexin in morphine dependence and withdrawal. J. Neurosci. 23, 3106-3111 (2003).
36. Paxinos, G. & Franklin, K.B.J. The mouse brain in stereotaxic coordinates (Academic Press, New York, 2000).