Intracisternal rSV40 administration provides effective pan-CNS transgene expression

Gene Therapy

Volumn 19, Issue 1, 2012, Pages 114-118

  Louboutin, J P ^a   Reyes, B A S ^a   Agrawal, L ^a   Van Bockstaele, E J ^a   Strayer, D S ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

Author keywords

cisterna magna; Cu Zn superoxide dismutase; gene transfer; gp120; HIV 1; neuroAIDS

Indexed keywords

ANTIOXIDANT; COPPER ZINC SUPEROXIDE DISMUTASE; GLUTATHIONE PEROXIDASE; GLYCOPROTEIN GP 120; MANNITOL; RECOMBINANT SV40 VECTOR; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; APOPTOSIS; ARTICLE; CENTRAL NERVOUS SYSTEM; CISTERNA MAGNA; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; GENE TRANSFER; INJECTION; NERVE CELL; NEUROPROTECTION; NONHUMAN; OXIDATIVE STRESS; PRIORITY JOURNAL; RAT; SIMIAN VIRUS 40; SUSTAINED DRUG RELEASE; TRANSGENICS; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; APOPTOSIS; CENTRAL NERVOUS SYSTEM; FEMALE; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLUTATHIONE PEROXIDASE; HIV ENVELOPE PROTEIN GP120; HIV INFECTIONS; HIV-1; IMMUNOHISTOCHEMISTRY; MANNITOL; NEURONS; NEUROPROTECTIVE AGENTS; OXIDATIVE STRESS; RATS; RATS, SPRAGUE-DAWLEY; SIMIAN VIRUS 40; SUPEROXIDE DISMUTASE; TRANSGENES;

HUMAN IMMUNODEFICIENCY VIRUS 1; RATTUS;

EID: 84855421063     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.75     Document Type: Article

References (38)

1. Akli S, Caillaud C, Vigne E, Stratford-Perricaudet LD, Poenaru L, Perricaudet M et al. Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 1993; 3: 224-228. (Pubitemid 23096557)
2. Betz AL, Shakui P, Davidson BL. Gene transfer to rodent brain with recombinant adenoviral vectors: effects of infusion parameters, infectious titer, and virus concentration on transduction volume. Exp Neurol 1998; 150: 136-142. (Pubitemid 28146432)
3. Wu P, Phillips MI, Bui J, Terwilliger EF. Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets. J Virol 1998; 72: 5919-5926. (Pubitemid 28283380)
4. McCown TJ, Xiao X, Li J, Breese GR, Samulski RJ. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res 1996; 713: 99-107. (Pubitemid 26114833)
5. Naldini L, Blomer U, Gage FH, Trono D, Verma IM. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 1996; 93: 11382-11388. (Pubitemid 26347130)
6. Kordower JH, Emborg ME, Bloch J, Ma SY, Chu Y, Leventhal L et al. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 2000; 290: 767-773.
7. Bosch A, Perret E, Desmaris N, Trono D, Heard JM. Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum Gene Ther 2000; 11: 1139-1150. (Pubitemid 30331580)
8. Skorupa AF, Fisher KJ, Wilson JM, Parente MK, Wolfe JH. Sustained production of betaglucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. Exp Neurol 1999; 160: 17-27.
9. Louboutin JP, Reyes BAS, Agrawal L, van Bockstaele EJ, Strayer DS. Strategies for CNS-directed gene delivery: in vivo gene transfer to the brain using SV40-derived vectors. Gene Therapy 2007; 14: 939-949. (Pubitemid 46865505)
10. Louboutin JP, Marusich E, Fisher-Perkins J, Dufour J, Bunnell BA, Strayer DS. Gene transfer to the Rhesus monkey brain using SV40-derived vectors is durable and safe. Gene Therapy 2011; e-pub ahead of print 3 March 2011; doi:10.1038/gt.2011.13.
11. Louboutin JP, Chekmasova AA, Marusich E, Chowdhury JR, Strayer DS. Efficient CNS gene delivery by intravenous injection. Nat Methods 2010; 7: 905-907.
12. Louboutin JP, Agrawal L, Reyes BAS, van Bockstaele EJ, Strayer DS. Protecting neurons from HIV-1 gp120-induced oxidant stress using both localized intracerebral and generalized intraventricular administration of antioxidant enzymes delivered by SV40-derived vectors. Gene Therapy 2007; 14: 1650-1661. (Pubitemid 350143190)
13. Agrawal L, Louboutin JP, Reyes BAS, van Bockstaele EJ, Strayer DS. Antioxidant enzyme gene delivery to protect from HIV-1 gp120-induced neuronal apoptosis. Gene Therapy 2006; 13: 1645-1656. (Pubitemid 44774209)
14. Agrawal L, Louboutin JP, Strayer DS. Preventing HIV-1 Tat-induced neuronal apoptosis using antioxidant enzymes: mechanistic and therapeutic implications. Virology 2007; 363: 462-472. (Pubitemid 46764954)
15. Louboutin JP, Reyes BAS, Agrawal L, van Bockstaele EJ, Strayer DS. HIV-1 gp120-induced neuroinflammation: relationship to neuron loss and protection by rSV40-delivered antioxidant enzymes. Exp Neurol 2010; 221: 231-245.
16. Louboutin JP, Reyes BAS, Agrawal L, van Bockstaele EJ, Strayer DS. Blood-brain barrier abnormalities caused by exposure to HIV-1 gp120-protection by gene delivery of antioxidant enzymes. Neurobiol Dis 2010; 38: 313-325.
17. Louboutin JP, Agrawal L, Reyes BAS, van Bockstaele EJ, Strayer DS. HIV-1 gp120-induced injury to the blood-brain barrier: role of metalloproteinases 2 and 9 and relationship to oxidative stress. J Neuropathol Exp Neurol 2010; 69: 801-816.
18. Turchan J, Pocernich CB, Gairola C, Chauhan A, Schifitto G, Butterfield DA et al. Oxidative stress in HIV demented patients and protection ex vivo with novel antioxidants. Neurology 2003; 60: 307-314. (Pubitemid 36152028)
19. McArthur JC, Brew BJ, Nath A. Neurological complications of HIV infection. Lancet Neurol 2005; 4: 543-555. (Pubitemid 41139976)
20. Mattson MP, Haughey NJ, Nath A. Cell death in HIV dementia. Cell Death Diff 2005; 12: 893-904. (Pubitemid 41030604)
21. Strayer DS. Gene therapy using SV40-derived vectors: what does the future hold? J Cell Physiol 1999; 181: 375-384. (Pubitemid 29513406)
22. Cordelier P, Calarota SA, Pomerantz RJ, Xiaoshan J, Strayer DS. Inhibition of HIV-1 in the central nervous system by IFN-alpha2 delivered by an SV40 vector. J Interferon Cytokine Res 2003; 23: 477-488. (Pubitemid 37130071)
23. Cordelier P, Van Bockstaele E, Calarota SA, Strayer DS. Inhibiting AIDS in the central nervous system: gene delivery to protect neurons from HIV. Mol Ther 2003; 7: 801-810. (Pubitemid 36790257)
24. Cordelier P, Strayer DS. Using gene delivery to protect HIV-susceptible CNS cells: inhibiting HIV replication in microglia. Virus Res 2006; 118: 87-97.
25. Ghodsi A, Stein C, Derksen T, Martins I, Anderson RD, Davidson BL. Systemic hyperosmolarity improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer. Exp Neurol 1999; 160: 109-116.
26. Fu H, Muenzer J, Samulski RJ, Breese G, Sifford J, Zeng X et al. Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther 2003; 6: 911-917. (Pubitemid 38072303)
27. Mastakov MY, Baer K, Xu R, Fitzsimons H, During MJ. Combined injection of rAAV with mannitol enhances gene expression in the rat brain. Mol Ther 2001; 3: 225-232. (Pubitemid 32509825)
28. Bourgoin C, Emiliani C, Kremer EJ, Gelot A, Tancini B, Gravel RA et al. Widespread distribution of b-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol. Gene Ther 2003; 10: 1841-1849. (Pubitemid 37185142)
29. Burger C, Nguyen FN, Deng J, Mandel RJ. Systemic mannitol-induced hyperosmolarity amplifies rAAV2-mediated striatal transduction to a greater extent than local coinfusion. Mol Ther 2005; 11: 327-331.
30. Nakane H, Chu Y, Faraci FM, Oberley LW, Heistad DD. Gene transfer of extracellular superoxide dismutase increases superoxide dismutase activity in cerebrospinal fluid. Stroke 2001; 32: 184-189. (Pubitemid 32063844)
31. Watanabe Y, Chu Y, Andresen JJ, Nakane H, Faraci FM, Heistad DD. Gene transfer of extracellular superoxide dismutase reduces cerebral vasospasm after subarachnoid hemorrhage. Stroke 2003; 34: 434-440. (Pubitemid 36206345)
32. Yamaguchi M, Zhou C, Heistad DD, Watanabe Y, Zhang JH. Gene transfer of extracellular superoxide dismutase failed to prevent cerebral vasospasm after experimental subarachnoid hemorrhage. Stroke 2004; 35: 2512-2517. (Pubitemid 39458409)
33. Shimamura M, Sato N, Oshima K, Aoki M, Kurinami H, Waguri S et al. Novel therapeutic strategy to treat brain ischemia. Overexpression of hepatocyte growth factor gene reduced ischemic injury without cerebral edema in rat model. Circulation 2004; 109: 424-431. (Pubitemid 38133914)
34. Strayer DS, Kondo R, Milano J, Duan LX. Use of SV40-based vectors to transduce foreign genes to normal human peripheral blood mononuclear cells. Gene Ther 1997; 4: 219-225. (Pubitemid 27182075)
35. Strayer DS, Lamothe M, Wei D, Milano J, Kondo R. Generation of recombinant SV40 vectors for gene transfer. SV40 protocols. In: Raptis L (ed). Methods in Molecular Biology, vol. 165. Humana Press: Totowa, NJ, 2001, pp 103-117.
36. Sauter BV, Parashar B, Chowdhury NR, Kadakol A, Ilan Y, Singh H et al. A replicationdeficient rSV40 mediates liver-directed gene transfer and a long-term amelioration of jaundice in gunn rats. Gastroenterology 2000; 119: 1348-1357.
37. Nosheny RL, Bachis A, Acquas E, Mocchetti I. Human immunodeficiency virus type 1 glycoprotein gp120 reduces the levels of brain-derived neurotrophic factor in vivo: potential implication for neuronal cell death. Eur J Neurosci 2004; 20: 2857-2864. (Pubitemid 40007279)
38. Mandel RJ, Rendahl KG, Spratt SK, Snyder RO, Cohen LK, Leff SE. Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease. J Neurosci 1998; 18: 4271-4284. (Pubitemid 28243820)