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Gene Technology Based Therapies in the Brain

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Advances and Technical Standards in Neurosurgery

Part of the book series: Advances and Technical Standards in Neurosurgery ((NEUROSURGERY,volume 31))

Abstract

Gene therapy potentially represents one of the most important developments in modern medicine. Gene therapy, especially of cancer, has created exciting and elusive areas of therapeutic research in the past decade. In fact, the first gene therapy performed in a human was not against cancer but was performed to a 14 year old child suffering from adenosine deaminase (ADA) deficiency. In addition to cancer gene therapy there are many other diseases and disorders where gene therapy holds exciting and promising opportunities. These include amongst others gene therapy within the central nervous system and the cardiovascular system. Improvements of the efficiency and safety of gene therapy is the major goal of gene therapy development. After the death of Jesse Gelsinger, the first patient in whom death could be directly linked to the viral vector used for the treatment, ethical doubts were raised about the feasibility of gene therapy in humans. Therefore, the ability to direct gene transfer vectors to specific target cells is also a crucial task to be solved and will be important not only to achieve a therapeutic effect but also to limit potential adverse effects.

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References

  1. Abeloff MD et al (2000) Clinical oncology, 2nd edn. Churchill, Livinstone

    Google Scholar 

  2. Alisky JA, Davidson BL (2000) Gene therapy for amyotrophic lateral sclerosis and other motor neuron diseases. Hum Gene Ther 11(17): 2315–2330

    Article  CAS  PubMed  Google Scholar 

  3. Ammirati M et al (1987) Effect of the extent of surgical resection on survival and quality of life in patients with supratentorial glioblastomas and anaplastic astrocytomas. Neurosurgery 21(2): 201–206

    CAS  PubMed  Google Scholar 

  4. Anderson DM et al (2003) Stability of mRNA/cationic lipid lipoplex in human and rat cerebrospinal fluid: methods and evidence for non-viral mRNA gene delivery into the central nervous system. Hum Gene Ther 14(3): 191–202

    Article  CAS  PubMed  Google Scholar 

  5. Ascadi G et al (2002) Increased survival and function of SOD 1 mice after glial-cell derived neurotrophic factor gene therapy. Hum Gene Ther 13(9): 1047–1059

    Google Scholar 

  6. Azzous M et al (2000) Increased motoneuron survival and improved neuromuscular function in transgenic ALS mice after intraspinal injection of adeno-associated virus encoding Bcl-2. Hum Mol Genet 9(5): 803–811

    Google Scholar 

  7. Azzous M et al (2004) VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 429: 413–417

    Google Scholar 

  8. Bachoud-Levi AC et al (2000) Neuroprotective gene therapy for Huntington’s disease using a polymer encapsulated BHK cell line engineered to secrete human CNTF. Hum Gene Ther 11(12): 1723–1729

    Article  CAS  PubMed  Google Scholar 

  9. Bachoud-Levi AC et al (1998) Prospective for cell and gene therapy in Huntington’s disease. Prog Brain Res 117: 511–524

    CAS  PubMed  Google Scholar 

  10. Bemelmans AP et al (1999) Brain-derived neurotrophic factors-mediated protection of striatal neurons in an excitotoxic rat model of Huntington’s disease, demonstrated by adenoviral gene transfer. Hum Gene Ther 10(18): 2987–2997

    Article  CAS  PubMed  Google Scholar 

  11. Bethel A et al (1997) Intravenous basic fibroblast growth factor-2 decreases brain injury resulting from focal ischemia in cats. Stroke 28: 609–615

    CAS  PubMed  Google Scholar 

  12. Bobo RH et al (1994) Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci 91: 2076–2080

    CAS  PubMed  Google Scholar 

  13. Boillée S and Cleveland DW (2004) Gene therapy for ALS delivers. Trends Neurosci 27(5): 235–238

    PubMed  Google Scholar 

  14. Bowers WJ et al (2003) Immune responses to replication-defective HSV-1 type vectors within the CNS: implication for gene therapy. Gene Ther 10(11): 941–945

    Article  CAS  PubMed  Google Scholar 

  15. Brown M (2003) Gene therapy success for Alzheimer’s. Drug Discov Today 8(11): 474–5

    PubMed  Google Scholar 

  16. Brown MD et al (2001) Gene delivery with synthetic (non-viral) carriers. Int J Pharm 229: 1–21

    Article  CAS  PubMed  Google Scholar 

  17. Bruijn LI et al (2004) Unraveling the mechanisms involved in motor neuron degeneration in ALS. Annu Rev Neurosci 27: 723–749

    Article  CAS  PubMed  Google Scholar 

  18. Byrnes AP et al (1995) Adenovirus gene transfer causes inflammation in the brain. Neuroscience 66(4): 1015–1024

    Article  CAS  PubMed  Google Scholar 

  19. Cabrera-Salazar MA et al (2002) Gene therapy for the lysosomal storage disorders. Curr Opin Mol Ther 4(4): 349–358

    CAS  PubMed  Google Scholar 

  20. Cavazzana-Calvo M et al (2000) Gene therapy of a human severe combined immunodeficiency (SCID)-XI disease. Science 288: 669–672

    Article  CAS  PubMed  Google Scholar 

  21. Chiocca EA (2003) Gene therapy: a primer for neurosurgens. Neurosurgery 53(2): 364–373

    PubMed  Google Scholar 

  22. Coffin M (1990) Retroviridae and their replication. In: Fields BN, Knipe DM (eds) Virology. Raven Press Ltd, New York, p 1437–1500

    Google Scholar 

  23. Connors TA (1995) The choice of prodrugs for gene directed enzyme prodrug therapy of cancer. Gene Ther 2: 702–709

    CAS  PubMed  Google Scholar 

  24. Cordelier P et al (2003) Inhibition of HIV-1 in the central nervous system by IFN-alpha2 delivered by an SV40 vector. J Interferon Cytokin Res 14(16): 1525–1533

    Google Scholar 

  25. Da Cruz MT et al (2004) Improving lipoplex-mediated gene transfer into C6 glioma cells and primary neurons. Exp Neurol 187(1): 65–75

    PubMed  Google Scholar 

  26. Datta K et al (2004) Senzitazing glioma cells to cisplatin by abrogating the p53 response with antisense oligonucleotides. Cancer Gene Ther 11(8): 525–531

    Article  CAS  PubMed  Google Scholar 

  27. Dietrich HH and Dacey RG (2000) Molecular keys to the problems of cerebral vasospasm. Neurosurgery 46(3): 517–29

    CAS  PubMed  Google Scholar 

  28. Durin MJ et al (2003) Development and optimization of adeno-associated virus vector transfer into the central nervous system. Methods Mol Med 76: 221–236

    Google Scholar 

  29. During MJ et al (1994) Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266: 1399–403

    CAS  PubMed  Google Scholar 

  30. Eberhardt O, Schulz JB (2004) Gene therapy in Parkinson’s disease. Cell Tissue Res 63(4): 758–769

    CAS  Google Scholar 

  31. Eberling JL et al (2003) In vivo PET imaging of gene expression in parkinsonian monkeys. Mol Ther 8(6): 873–5

    Article  CAS  PubMed  Google Scholar 

  32. Edelstein ML et al (2004) Gene therapy clinical trials worldwide 1989–2004 — an overview. J Gene Med 6(6): 597–602

    Article  PubMed  Google Scholar 

  33. Ehrengruber MU (2002) Alphaviral vectors for gene transfer into neurons. Mol Neurobiol 26: 183–201

    CAS  PubMed  Google Scholar 

  34. Eto Y, Ohashi T (2002) Novel treatment for neuronopathic lysosomal storage diseases — cell therapy/gene therapy. Curr Mol Med 2(1): 83–89

    Article  CAS  PubMed  Google Scholar 

  35. Fazakerley JK (2004) Semliki forest virus infection of laboratory mice: a model to study the pathogenesis of viral encephalitis. Arch Virol Suppl 18: 179–190

    PubMed  Google Scholar 

  36. Fecci PE, Gromeier M, Sampson JH (2000) Viruses in the treatment of brain tumors. Neuroimaging Clin N Am 12(4): 553–570

    Google Scholar 

  37. Ferrari FK et al (1997) New developments in the generation of Ad-free, high titer rAAV gene theraby vectors. Nat Med 3(11): 1295–1297

    Article  CAS  PubMed  Google Scholar 

  38. Finkelstein R, Baughman RW, and Steele FR (2001) Harvesting the neural gene therapy fruit. Mol Ther 3(1): 3–7

    Article  CAS  PubMed  Google Scholar 

  39. Fisher W (1987) Amelioration of cholinergic neuron atrophy and spatial memory impairment in aged rats by nerve growth factor. Nature 329: 65–68

    Google Scholar 

  40. Friedlos F et al (1998) Gene directed enzyme prodrug therapy: quantitative bystander cytotoxicity and DNA damage induced by CB 1954 in cells expressing bacterial nitroreductase. Gene Ther 5(1): 105–112

    Article  CAS  PubMed  Google Scholar 

  41. Friese MA et al (2003) MICA/NKG2D-mediated immunogene therapy of experimental gliomas. Cancer Res 63(24): 8996–9006

    CAS  PubMed  Google Scholar 

  42. Futerman AH, van Meer G (2004) The cell biology of lysosomal storage disorders. Nat Rev Mol Cell Biol 5(7): 554–565

    Article  CAS  PubMed  Google Scholar 

  43. Gash DM, Gerhardt GA, Hoffer BJ (1998) Effects of glial cell line-derived neurotrophic factor on the nigrostriatal dopamine system in rodents and nonhuman primates. Adv Pharmacol 42: 911–915

    CAS  PubMed  Google Scholar 

  44. Ge L et al (1995) Gene therapeutic approaches to primary and metastatic brain tumors: II. Ribozyme-mediated suppression of CD44 expression. J Neurooncol 26(3): 251–257

    Article  CAS  PubMed  Google Scholar 

  45. Goldman CK et al (1997) In vitro and in vivo gene delivery mediated by a synthetic polycationic amino polymer. Nat Biotech 15(5): 462–466

    Article  CAS  Google Scholar 

  46. Gomez-Manzano C et al (2004) A novel E1A-E1B mutant adenovirus induces glioma regression in vivo. Oncogene 23(10): 1821–1828

    Article  CAS  PubMed  Google Scholar 

  47. Gondi CS et al (2004) Adenovirus-mediated expression of antisense urokinase plasminogen activator receptor and antisense cathepsin B inhibits tumor growth, invasion, and angiogenesis in gliomas. Cancer Res 64: 4069–4077

    Article  CAS  PubMed  Google Scholar 

  48. Greg M (2002) Breaking down barriers. Science 297(5587): 1116–1118

    Google Scholar 

  49. Gromeier M, Wimmer E (2001) Viruses for the treatment of malignant glioma. Curr Opin Mol Ther 3(5): 503–508

    CAS  PubMed  Google Scholar 

  50. Gutierrez-Delicado E, Serratosa JM (2004) Genetics of epilepsies. Curr Opin Neurol 17(2): 147–153

    Article  CAS  PubMed  Google Scholar 

  51. Haberman RP et al (2003) Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med 9(8): 1076–1080

    Article  CAS  PubMed  Google Scholar 

  52. Hacein-Bey-Abina S et al (2003) A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348(3): 255–6

    Article  PubMed  Google Scholar 

  53. Hagell P et al (2002) Dyskinesias following neural transplantation in Parkinsons disease. Nat Neurosci 5: 627–628

    CAS  PubMed  Google Scholar 

  54. Haluska M, Anthony ML (2004) Osmotic blood-brain barrier modification for the treatment of malignant brain tumors. CJON 8(3): 263–267

    Google Scholar 

  55. Hoehn B et al (2001) Overexpression of HSP72 after induction of experimental stroke protects neurons from ischemic damage. J Cereb Blood Flow Metab 21(11): 1303–1309

    CAS  PubMed  Google Scholar 

  56. Hogarth P (2003) Huntington’s disease: a decade beyond gene discovery. Curr Neurol Neurosci Rep 3(4): 279–284

    PubMed  Google Scholar 

  57. Howard K (2003) First Parkinson gene therapy trial launches. Nat. Biotech 21(10): 1117–8

    Article  CAS  Google Scholar 

  58. Hsich G, Sena-Esteves M, Breakefield XO (2002) Critical issues in gene therapy for neurologic disease. Hum Gene Ther 13(5): 579–604

    Article  CAS  PubMed  Google Scholar 

  59. Ichikawa T et al (2001) Intraneoplastic polymer-based delivery of cyclophosphamide for intratumoral bioconversion by a replicating oncolytic viral vector. Cancer Res 61(3): 864–868

    CAS  PubMed  Google Scholar 

  60. Immonen A et al (2004) AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomized, controlled study. Mol Ther 10(5): 967–72

    Article  CAS  PubMed  Google Scholar 

  61. Jakobsson J et al (2003) Targeted transgene expression in rat brain using lentiviral vectors. J Neurosci Res 73(6): 876–885

    Article  CAS  PubMed  Google Scholar 

  62. Jakobsson J et al (2003) Targeted transgene expression in rat using lentiviral vectors. J Neurosci Res 73(6): 876–885

    Article  CAS  PubMed  Google Scholar 

  63. Joos K, Chirmule N (2003) Immunity to adenovirus and adenoassociated viral vectors: implications for gene therapy. Gene Ther 10(11): 955–963

    Google Scholar 

  64. Kajiwara K et al (2000) Humoral immune response to adenovirus vectors in the brain. J Neuroimmunol 103(3): 253–265

    Google Scholar 

  65. Kaspar BK et al (2003) Retrograde viral delivery of IGF-1 prolongs survival in a mouse model. Science 301: 839–842

    Article  CAS  PubMed  Google Scholar 

  66. Kaye EM, Sena-Esteves M (2002) Gene therapy for the central nervous system in the lysosomal storage disorders. Neurol Clin 20(3): 879–901

    Article  PubMed  Google Scholar 

  67. Kirik D, Bjorklund A (2003) Modeling CNS neurodegeneration by overexpression of disease causing proteins using viral vectors. Trends Neurosci 26(7): 386–392

    Article  CAS  PubMed  Google Scholar 

  68. Kirsch M et al (2000) Anti-angiogenic treatment strategies for malignant brain tumors. J Neurooncol 50: 149–163

    CAS  PubMed  Google Scholar 

  69. Koponen JK et al (2003) Doxycycline-regulated lentiviral vector system with a novel reverse transactivator rtTA2S-M2 shows a tight control of gene expression in vitro and in vivo. Gene Ther 10(6): 459–466

    Article  CAS  PubMed  Google Scholar 

  70. Kordower J et al (2000) Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson’s disease. Science 290: 767–773

    Article  CAS  PubMed  Google Scholar 

  71. Kramm CM et al (1996) Long-term survival in a rodent model of disseminated brain tumors by combined intrathecal delivery of herpes vectors and ganciclovir treatment. Hum Gene Ther 7(16): 1989–1994

    CAS  PubMed  Google Scholar 

  72. Kromer LF (1987) Nerve growth factor treatment after brain injury prevents neuronal death. Science 235(4785): 214–216

    CAS  PubMed  Google Scholar 

  73. Kugler S et al (2003) Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area. Gene Ther 10(4): 337–347

    CAS  PubMed  Google Scholar 

  74. Lakka SS et al (2003) Synergistic down-regulation of urokinase plasminogen activator receptor and matrix metalloproteinase-9 in SNB19 glioblastoma cells efficiently inhibits glioma cell invasion, angiogenesis, and tumor growth. Cancer Res 63(10): 2454–2461

    CAS  PubMed  Google Scholar 

  75. Lamont JP et al (2000) A prospective phase II trial of ONYX-015 adenovirus and chemotherapy in recurrent squamous cell carcinoma of the head and neck (the Baylor experience). Ann Surg Oncol 7(8): 588–592

    CAS  PubMed  Google Scholar 

  76. Lampela P et al (2002) The use of low-molecular-weight PEIs as gene carriers in the monkey fibroblastoma and rabbit smooth muscle cell culture. J Gene Med 4(2): 205–214

    Article  PubMed  Google Scholar 

  77. Lehtolainen P et al (2002) Baculovirus exhibits restricted cell type specificity in rat brain: a comparison of baculovirus-and adenovirus mediated intracerebral gene transfer in vivo. Gene Ther 9(24): 1693–1699

    Article  CAS  PubMed  Google Scholar 

  78. Lehtolainen P et al (2002) Clining and characterization of Scavidin, a fusion protein for the targeted delivery of biotinylated molecules. J Biol Chem 277(10): 8545–8550

    Article  CAS  PubMed  Google Scholar 

  79. Lehtolainen P et al (2003) Targeting of biotinylated compounds to its target tissue using a low-density lipoprotein receptor-avidin fusion protein. Gene Ther 10(25): 2090–2097

    Article  CAS  PubMed  Google Scholar 

  80. Lesage D et al (2002) Evaluation and optimization on DNA delivery into gliosarcoma 9L cells by a cholesterol-based cationic liposome. Biochim Biophys Acta 1564(2): 393–402

    CAS  PubMed  Google Scholar 

  81. Lin H et al (2002) Cyclooxygenase-1 and bicistronic cyclooxygenase-1/prostacyclin synthase gene transfer protects against ischemic cerebral infarction. Circulation 105(16): 1962–1969

    Article  CAS  PubMed  Google Scholar 

  82. Liu BH et al (2004) CMV enhancer/human PDGF-beta promoter for neuron specific transgene expression. Gene Ther 11(1): 52–60

    Article  PubMed  Google Scholar 

  83. Lou E (2004) Oncolytic viral therapy and immunotherapy of malignant brain tumors: two potential new approaches of translational research. Ann Med 36(1): 2–8

    CAS  PubMed  Google Scholar 

  84. Lowenstein PR, Castro MG (2003) inflammation and adaptive immune responses to adenoviral vectors injected into the brain: peculiarities, mechanisms, and consequences. Gene Ther 10(11): 946–954

    CAS  PubMed  Google Scholar 

  85. Lowenstein PR (2004) Immunological needles in the gene therapy haystack: applying a genetic paradigm to gene therapy. Gene Ther 11(1): 1–3

    Article  CAS  PubMed  Google Scholar 

  86. Lüders JC et al (2000) Adenoviral gene transfer of nitric oxide synthase increases cerebral blood flow in rats. Neurosurgery 47(5): 1206–1217

    PubMed  Google Scholar 

  87. Lundstrom K et al (2001) Semliki Forest virus vectors: efficient vehicles for in vitro and in vivo gene delivery. FEBS Lett 504(3): 99–103

    Article  CAS  PubMed  Google Scholar 

  88. Luo J et al (2002) Subthalamic GAD gene therapy in a Parkinson’s disease rat model. Science 298: 425–429

    Article  CAS  PubMed  Google Scholar 

  89. MacMillan JC et al (1994) Clinical considerations in gene therapy of Huntington’s disease. Gene Ther 1[Suppl] 1: 88

    Google Scholar 

  90. McCown TJ (2004) The clinical potential of antiepileptic gene therapy. Expert Opin Biol Ther 4(11): 1771–1776

    Article  CAS  PubMed  Google Scholar 

  91. McPhee SW et al (2005) Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease. Brain Res Mol Brain Res 135(1–2): 112–121

    CAS  PubMed  Google Scholar 

  92. Manome Y et al (1996) Gene therapy for malignant gliomas using replication incompetent retroviral and adenoviral vectors encoding the cytochrome P450 2B1 gene together with cyclophosphamide. Gene Ther 3(6): 513–520

    CAS  PubMed  Google Scholar 

  93. Markert JM et al (2000) Conditionally replicating herpes simplex virus mutant, GS07 for the treatment of malignant glioma: results of a phase I trial. Gene Ther 7(10): 867–874

    Article  CAS  PubMed  Google Scholar 

  94. Marr RA et al (2003) Neprilysin gene transfer reduces human amyloid pathology in transgenic mice. J Neurosci 23(6): 1992–1996

    CAS  PubMed  Google Scholar 

  95. Marr RA et al (2004) Neprilysin regulates amyloid Beta peptide levels. J Mol Neurosci 22(1–2): 5–11

    PubMed  Google Scholar 

  96. Mattson MP (2004) Pathways towards and away from Alzheimer’s disease. Nature 430: 631–639

    Article  CAS  PubMed  Google Scholar 

  97. McBride JL et al (2003) Structural and functional neuroprotection in a rat model of Huntington’s disease by viral gene transfer of GDNF. Exp Neurol 181(2): 213–223

    Article  CAS  PubMed  Google Scholar 

  98. Miller CR et al (1998) Differential susceptibility of primary and established human glioma cells to adenovirus infection: targeting via the epidermal growth factor receptor achieves fibre receptor-independent gene transfer. Cancer Res 58(24): 5738–5748

    CAS  PubMed  Google Scholar 

  99. Miller CR et al (2002) Intratumoral 5-fluorouracil produced by cytosine deaminase/5-fluorocytosin gene therapy is effective for experimental human glioblastoma. Cancer Res 62(3): 773–780

    CAS  PubMed  Google Scholar 

  100. Miller DG et al (1990) Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 10: 4239–4242

    CAS  PubMed  Google Scholar 

  101. Mohanam S et al (2002) Modulation of invasive properties of human glioblastoma cells stably expressing amino-terminal fragment of urokinase-type plasminogen activator. Oncogene 21(51): 7824–7830

    Article  CAS  PubMed  Google Scholar 

  102. Monahan PE, White GC 2nd (2002) Hemophilia gene therapy: update. Curr Opin Hematol 9(5): 430–436

    Article  PubMed  Google Scholar 

  103. Moolten FL (1994) Drug sensitivity (“suicide”) genes for selective cancer chemotherapy. Cancer Gene Ther 1: 279–287

    CAS  PubMed  Google Scholar 

  104. Morishita R et al (2004) Safety Evaluation of Clinical Gene Therapy Using Hepatocyte Growth Factor to Treat Peripheral Arterial Disease. Hypertension Jul 6 [Epub ahead of print]

    Google Scholar 

  105. Morishita R et al (2004) Therapeutic angiogenesis using hepatocyte growth factor (HGF). Curr Gene Ther 4(2): 199–206

    CAS  PubMed  Google Scholar 

  106. Moriuchi S et al (1998) Enhanced tumor cell killing in the presence of ganciclovir by herpes simplex virus type 1 vector-directed coexpression of human tumor necrosis factor-alpha and herpes simplex virus thymidine kinase. Cancer Res 58(24): 5731–5737

    CAS  PubMed  Google Scholar 

  107. Mountain A (2000) Gene therapy: the first decade. TIBTECH 18: 119–128

    CAS  Google Scholar 

  108. Naldini L et al (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263–267

    CAS  PubMed  Google Scholar 

  109. Neuwelt EA et al (1995) Gene replacement therapy in the central nervous system — viral vector-mediated therapy of global neurodegenerative disease. Behav Brain Sci 18(1): 1–9

    Google Scholar 

  110. Neuwelt G et al (1980) Reversible osmotic blood-brain barrier disruption in humans: implications for the chemotherapy of malignant brain tumors. Neurosurgery 7(1): 44–52

    CAS  PubMed  Google Scholar 

  111. Nguyen TT et al (2003) Convective distribution of macromolecules in the primate brain demonstrated using computerized tomography and magnetic resonance imaging. J Neurosurg 98(3): 584–590

    CAS  PubMed  Google Scholar 

  112. Ogris M, Wagner E (2002) Targeting tumors with non-viral gene delivery systems. Drug Discov Today 7(8): 479–485

    Article  CAS  PubMed  Google Scholar 

  113. Onoue H et al (1998) Expression and function of recombinant endothelial nitric oxide synthase gene in canine basilar artery after experimental subarachnoid hemorrhage. Stroke 29: 1959–1966

    CAS  PubMed  Google Scholar 

  114. Palmer AM et al (2002) Pharmacotherapy for Alzheimer’s disease: progress and prospects. Trends Pharmacol Sci 23: 426–433

    Article  CAS  PubMed  Google Scholar 

  115. Palmer DH et al (2004) Virus-directed enzyme prodrug therapy: intratumoral administration of a replication-deficient adenovirus encoding nitro-reductase to patients with resectable liver cancer. J Clin Oncol 22(9): 1546–1552

    Article  CAS  PubMed  Google Scholar 

  116. Pardridge WM (2002) Drug and gene delivery to the brain: the vascular route. Neuron 36: 555–558

    Article  CAS  PubMed  Google Scholar 

  117. Pardridge WM (2002a) drug and gene delivery to the brain: the vascular route. Neuron 36(4): 555–558

    Article  CAS  PubMed  Google Scholar 

  118. Pardridge WM (2002b) Drug and gene targeting to the brain with molecular Trojan horses. Nat Rev Drug Discov 1(2): 131–139

    Article  CAS  PubMed  Google Scholar 

  119. Pompl PN et al (2003) A therapeutic role for cyclooxygenase-2 inhibitors in a transgenic mouse model of amyotrophic lateral sclerosis. FASEB 17(6): 725–727

    CAS  Google Scholar 

  120. Prados MD et al (2003) Treatment of progressive or recurrent glioblastoma multiforme in adults with herpes simplex virus thymidine kinase gene vector-producer cells followed by intravenous ganciclovir administration: a phase I/II multi-institutional trial. J Neurooncol 65(3): 269–278

    Article  PubMed  Google Scholar 

  121. Puduvalli VK (2004) Inhibition of angiogenesis as a therapeutic strategy against brain tumors. Cancer Treat Res 117: 307–336

    CAS  PubMed  Google Scholar 

  122. Puumalainen AM et al (1998b) Beta-galactosidase gene transfer to human malignant glioma in vivo using replication-deficient retroviruses and adenoviruses. Hum Gene Ther 9(12): 1769–1774

    CAS  PubMed  Google Scholar 

  123. Puumalainen AM, Vapalahti M, Ylä-Herttuala S (1998a) Gene therapy for malignant glioma patients. Adv Exp Med Biol 451: 505–509

    CAS  PubMed  Google Scholar 

  124. Qureshi NH et al (2000) Multicolumn infusion of gene therapy cells into human brain tumors: technical report. Neurosurgery 46(3): 663–668; discussion 668–669

    CAS  PubMed  Google Scholar 

  125. Rabino I (2003) Gene therapy: ethical issues. Theor Med 24: 31–58

    Google Scholar 

  126. Rainov NG (2000a) A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Hum Gene Ther 11(17): 2389–2401

    Article  CAS  PubMed  Google Scholar 

  127. Rainov NG et al (2000b) Immune response induced by retrovirus-mediated HSV-tk/GCV pharmacogene therapy in patient with glioblastoma multiforme. Gene Ther 7(21): 1853–1858

    Article  CAS  PubMed  Google Scholar 

  128. Rainov NG, Kramm CM (2001) Vector delivery methods and targeting strategies for gene therapy of brain tumors. Curr Gene Ther 1: 367–383

    Article  CAS  PubMed  Google Scholar 

  129. Rainov NG, Ren H (2003) Oncolytic viruses for treatment of malignant brain tumours. Acta Neurochir [Suppl] 88: 113–123

    CAS  Google Scholar 

  130. Rainov NG et al (1999) Intraarterial delivery of adenovirus vectors and liposome-DNA complexes to experimental brain neoplasms. Hum Gene Ther 10(2): 311–318

    Article  CAS  PubMed  Google Scholar 

  131. Regulier E et al (2002) Dose-dependent neuroprotective effect of ciliary neurotrophic factor delivered via tetracycline-regulated lentiviral vectors in the quinolinic acid rat model of Huntington’s disease. Hum Gene Ther 13(16): 1981–1990

    Article  CAS  PubMed  Google Scholar 

  132. Richichi C et al (2004) Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus. J Neurosci 24(12): 3051–3059

    Article  CAS  PubMed  Google Scholar 

  133. Saito R et al (2003) Distribution of liposomes into brain and rat brain tumor models by convection-enhanced delivery monitored with magnetic resonance imaging. Cancer Res 64: 2572–2579

    Google Scholar 

  134. Samii A, Nutt JG, Ransom BR (2004) Parkinson’s disease. Lancet 363: 1783–93

    Article  CAS  PubMed  Google Scholar 

  135. Sanchez-Pernaut R et al (2001) Functional effect of adeno-associated virus mediated gene transfer of aromatic L-amino acid decarboxylase into the striatum of 6-OHDA-lesioned rats. Mol Ther 4(4): 324–330

    Google Scholar 

  136. Sandmair A-M et al (2000a) Thymidine kinase gene therapy for human malignant glioma using replication-deficient retroviruses or adenoviruses. Human Gene Ther 11(16): 2197–2205

    Article  CAS  Google Scholar 

  137. Sandmair A-M (2000b) Gene therapy for malignant glioma. Thesis work. University of Kuopio

    Google Scholar 

  138. Schlachetzki F et al (2004) Gene therapy of the brain. Neurology 62: 1275–1281

    CAS  PubMed  Google Scholar 

  139. Scollay R (2001) Gene therapy: a brief overview of the past, present and future. Ann NY Acad Sci 953: 26–30

    CAS  PubMed  Google Scholar 

  140. Seki T et al (2004) Adenoviral gene transfer of aspartoacylase ameliorates tonic convulsions of spontaneously epileptic rats. Neurochem Int 45(1): 171–178

    Article  CAS  PubMed  Google Scholar 

  141. Shand N et al (1999) A phase 1–2 clinical trial of gene therapy for recurrent glioblastoma multiforme by tumor transduction with the herpes simplex thymidine kinase gene followed by ganciclovir. GLI328 European-Canadian Study Group. Hum Gene Therapy 10(14): 2325–2335

    CAS  Google Scholar 

  142. Shen Y et al (2000) Triple transduction with adeno-associated virus vectors expressing tyrosine hydroxylase, aromatic-L-amino-acid decarboxylase, and GTP cyclohydrolase I for gene therapy of Parkinson’s disease. Hum Gene Ther 11(11): 1509–1519

    Article  CAS  PubMed  Google Scholar 

  143. Shigeru T et al (2004) Infiltrating macrophages as in vivo targets for intravenous gene delivery in cerebral infarction. Stroke Published online before print

    Google Scholar 

  144. Shimamura M et al (2003) HJV-envelope vector for gene transfer into central nervous system. Biochem Biophys Res Commun 300(2): 464–471

    Article  CAS  PubMed  Google Scholar 

  145. Shimamura M et al (2004) Novel therapeutic strategy to treat brain ischemia: overexpression of hepatocyte growth factor gene reduced ischemic injury without cerebral edema in rat model. Circulation 109(3): 424–431

    Article  CAS  PubMed  Google Scholar 

  146. Shimoura N, Hamada H (2003) Gene therapy using an adenovirus vector for apoptosis-related genes is a highly effective therapeutic modality for killing glioma cells. Curr Gene Ther 3(2): 147–153

    Google Scholar 

  147. Shirakura M et al (2004) Postischemic administration of Sendai virus vector carrying neurotrophic factor genes prevents delayed neuronal death in gerbils. Gene Ther 11(9): 784–790

    Article  CAS  PubMed  Google Scholar 

  148. Simonato M et al (2000) Gene transfer into neurons for the molecular analysis of behaviour: focus on herpes simplex vectors. Trends Neurosci 23(5): 183–190

    Article  CAS  PubMed  Google Scholar 

  149. Smitt PS et al (2003) Treatment of relapsed malignant glioma with an adenoviral vector containing the herpes simplex thymidine kinase gene followed by ganciclovir. Mol Ther 7(6): 851–858

    Article  CAS  PubMed  Google Scholar 

  150. Snyder RO, Flotte TR (2002) Production of clinical grade recombinant adeno-associated virus vectors. Curr Opin Biotechnol 13(5): 418–423

    Article  CAS  PubMed  Google Scholar 

  151. Steinbach JP and Weller M (2002) Mechanisms of apoptosis in central nervous system tumor: application to theory. Curr Neurol Neurosci Rep 2(3): 246–253

    PubMed  Google Scholar 

  152. Steinwaerder DS et al (2001) Tumor-specific gene expression in hepatic metastases by a replication-activated adenovirus vector. Nat Med 7(2): 240–243

    Article  CAS  PubMed  Google Scholar 

  153. Stoodley M et al (2000) Effect of adenovirus-mediated nitric oxide synthase gene transfer on vasospasm after experimental subarachnoid hemorrhage. Neurosurgery 46(5): 1193–1205

    CAS  PubMed  Google Scholar 

  154. Sun JY et al (2003) Immune responses to adeno-associated virus and its recombinant vectors. Gene Ther 10(11): 964–976

    Article  CAS  PubMed  Google Scholar 

  155. Sun M et al (2003) Correction of a rat model of Parkinson’s disease by coexpression of tyrosine hydroxylase and aromatic amino acid decarboxylase from a helper virus-free herpes simplex virus type 1 vector. Hum Gene Ther 14(5): 415–424

    Article  CAS  PubMed  Google Scholar 

  156. Tanaka T et al (1998) Viral vector-targeted antiangiogenic gene therapy utilizing an agiostatin complementary DNA. Cancer Res 58(15): 3362–3369

    CAS  PubMed  Google Scholar 

  157. Tani H et al (2003) In vitro and in vivo gene delivery by recombinant Baculoviruses. J Virol 77(18): 9799–9808

    Article  CAS  PubMed  Google Scholar 

  158. Temin HM (1990) Safety considerations in somatic gene therapy of human disease with retroviral vectors. Human Gene Ther 1: 111–123

    CAS  Google Scholar 

  159. Tenenbaum L et al (2004) Recombinant AAV-mediated gene delivery to the central nervous system. J Gene Med 6: 212–222

    Article  Google Scholar 

  160. Thomas CE et al (2003) Progress and problems with the use of viral vectors for gene therapy. Nature 4: 346–358

    CAS  Google Scholar 

  161. Todo T et al (2000) Evaluation of ganciclovir-mediated enhancement of the antitumoral effect in oncolytic, multimediated herpes simplex virus type 1 (G207) therapy of brain tumors. Cancer Gene Ther 7(6): 939–946

    Article  CAS  PubMed  Google Scholar 

  162. Toyoda K et al (2003) Gene therapy for cerebral vascular disease: update 2003. Br J Pharmacol 139: 139–147

    Article  Google Scholar 

  163. Trask TW et al (2000) Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors. Mol Ther 1(2): 195–203

    Article  CAS  PubMed  Google Scholar 

  164. Tuszynski MH et al (1998) Targeted intraparenchymal delivery of human NGF by gene transfer to the primate forebrain for 3 months does not accelerate β-amyloid plaque deposition. Exp Neurol 154: 573–582

    Article  CAS  PubMed  Google Scholar 

  165. Tuszynski MH (2002) Growth-factor gene therapy for neurodegenerative disorders. Lancet Neurol 1: 51–57

    PubMed  Google Scholar 

  166. Uchida H et al (2004) Adenovirus-mediated transfer of siRNA against surviving induced apoptosis and attenuated tumor cell growth in vitro and in vivo. Mol Ther 10(1): 162–171

    Article  CAS  PubMed  Google Scholar 

  167. Van der Haute C et al (2003) Lentiviral vector-mediated delivery of short hairpin RNA results in persistent knockdown of gene expression in mouse brain. Hum Gene Ther 14(18): 1799–1807

    PubMed  Google Scholar 

  168. Wakimoto H et al (2003) Effects of innate immunity on herpes simplex virus and its ability to kill tumor cells. Gene Ther 10(11): 983–990

    Article  CAS  PubMed  Google Scholar 

  169. Wang LJ et al (2002) Neuroprotective effects of glial cell-line derived neurotrophic factor mediated by adeno-associated virus in a transgenic animal model of amyotrophic lateral sclerosis. J Neurosci 22(16): 6920–6928

    CAS  PubMed  Google Scholar 

  170. Watanabe Y et al (2003) Gene transfer of extracellular superoxide dismutase reduces cerebral vasospasm after subarachnoid hemorrhage. Stroke 34(2): 434–440

    Article  CAS  PubMed  Google Scholar 

  171. Watson DJ, Wolfe JH (2003) Lentiviral vectors for gene transfer to the central nervous system. Applications in lysosomal storage disease animal models. Methods Mol Med 76: 383–403

    CAS  PubMed  Google Scholar 

  172. Weedon SJ et al (2000) Sensitisation of human carcinoma cells to the prodrug CB1954 by adenovirus vector-mediated expression of E coli nitroreductase. Int J Cancer 86(6): 848–854

    Article  CAS  PubMed  Google Scholar 

  173. Weiss MD et al (2004) Current pharmacological management of amyotrophic [corrected] lateral sclerosis and a role rational polypharmacy. Expert Opin Pharmacother 5(4): 735–746.

    Article  PubMed  Google Scholar 

  174. Wickham TJ (2003) Ligand-directed targeting of genes to the site of disease. Nat Med 9(1): 135–139

    Article  CAS  PubMed  Google Scholar 

  175. Wildner O (1999) In situ use of suicide genes for therapy of brain tumours. Ann Med 31(6): 421–429

    CAS  PubMed  Google Scholar 

  176. Winkler J et al (1998) Cholinergic strategies for Alzheimer’s disease. J Mol Med 76: 555–567

    Article  CAS  PubMed  Google Scholar 

  177. Witham TF et al (2003) Expression of a soluble transforming growth factorbeta (TGFbeta) receptor reduces tumorigenicity by regulation natural killer (NK) cell activity against 9L gliosarcoma in vivo. J Neurooncol 64(1–2): 55–61

    Google Scholar 

  178. Wood MJ et al (1994) Immunological consequences of HSV-1-mediated gene transfer into the CNS. Gene Ther [Suppl] 1: 82

    Google Scholar 

  179. Wu K et al (2004) The effects of rAAV2-mediated NGF gene delivery in adult and aged rats. Mol Ther 9(2): 262–269

    Article  CAS  PubMed  Google Scholar 

  180. Yamanaka R et al (2003) Induction of antitumor immunological response by an intratumoral injection of dendritic cells pulsed with genetically engineered Semliki Forest virus to produce interleukin-18 combined with the systemic administration of interleukin-12. J Neurosurg 99(4): 746–753

    CAS  PubMed  Google Scholar 

  181. Yang GY et al (1997) Overexpression of interleukin-1 receptor antagonist in the mouse brain reduces ischemic brain injury. Brain Res 751(2): 181–188

    Article  CAS  PubMed  Google Scholar 

  182. Yang SY et al (2004) Gene therapy of rat malignant gliomas using neural stem stells expressing IL-12. DNA Cell Biol 23(6): 381–389

    Article  CAS  PubMed  Google Scholar 

  183. Yenari MA et al (2003) Gene therapy and hypothermia for stroke treatment. Ann N Y Acad Sci 992: 54–68; discussion 79–81

    Google Scholar 

  184. Ylä-Herttuala S, Alitalo K (2003) Gene transfer as a tool to induce therapeutic vascular growth. Nat Med 9(6): 694–701

    Article  PubMed  Google Scholar 

  185. Ylä-Herttuala S and Martin JF (2000) Cardiovascular gene therapy. Lancet 355: 213–222

    PubMed  Google Scholar 

  186. Yukawa H et al (2000) Adenoviral gene transfer of basic fibroblast growth factor promotes angiogenesis in rat brain. Gene Ther 7: 942–949

    Article  CAS  PubMed  Google Scholar 

  187. Zadeh G and Guha A (2003) Angiogenesis in nervous system disorders. Neurosurgery 53(6): 1362–1374

    PubMed  Google Scholar 

  188. Zhang Y et al (2004) Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain tumour. Clin Cancer Res 10(11): 3667–3677

    Article  CAS  PubMed  Google Scholar 

  189. Zlokovic BV and Apuzzo M (1997) Cellular and molecular neurosurgery: pathways from concept to reality — part II: vector systems and delivery methodologies for gene therapy of the central nervous system. Neurosurgery 40(4): 1992–1998

    Google Scholar 

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Authors and Affiliations

  1. A. I. Virtanen Institute, University of Kuopio, Kuopio, Finland

    T. Wirth & S. Ylä-Herttuala

  2. Department of Medicine, University of Kuopio, Kuopio, Finland

    S. Ylä-Herttuala

  3. Gene Therapy Unit, Kuopio University Hospital, Kuopio, Finland

    S. Ylä-Herttuala

  4. Ark Therapeutics Oy, Kuopio, Finland

    T. Wirth

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  1. T. Wirth
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  2. S. Ylä-Herttuala
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Editors and Affiliations

  1. Cambridge

    J. D. Pickard

  2. Ankara

    N. Akalan

  3. Roma

    C. Di Rocco

  4. Ljubljana

    V. V. Dolenc

  5. Erlangen

    R. Fahlbusch

  6. Lisbon

    J. Lobo Antunes

  7. Lyon

    M. Sindou

  8. Lausanne

    N. de Tribolet

  9. Utrecht

    C. A. F. Tulleken

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Wirth, T., Ylä-Herttuala, S. (2006). Gene Technology Based Therapies in the Brain. In: Pickard, J.D., et al. Advances and Technical Standards in Neurosurgery. Advances and Technical Standards in Neurosurgery, vol 31. Springer, Vienna. https://doi.org/10.1007/3-211-32234-5_1

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