Gene therapy for neurological disease

Bibliography

• BLESCH A, GRILL RJ,TUSZYNSKI MH: Neurotrophin gene therapy in models of CNS trauma and neurodegeneration. Frog. Brain Res. (1998) 117:473–484.
   PubMed Web of Science ®Google Scholar
• BRUSTLE O, MCKAY RD: Neuronal progenitors as tools for cell replacement in the nervous system. Curt: Opin. Neurobiol (1996) 6:688–695.
   PubMed Web of Science ®Google Scholar
• •An excellent review of thetherapeutic potential of stem-like cells for neurotherapy.
   Google Scholar
• MUZYCZKA N: Use of anedo-associated virus as a general transduction vector for mammalian cells. Curc Top. Microbiol Immunol (1992) 158:97–129.
   PubMed Web of Science ®Google Scholar
• NALDINI L, BLOMER U, GALLAY P et al.: hi vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science (1996) 272:263–267.
   PubMed Web of Science ®Google Scholar
• RABINOWITZ JE, SAMULSKI RJ: Building a better vector: the manipulation of AAV virions. Virology (2000) 278:301–308.
   PubMed Web of Science ®Google Scholar
• JACOBS A, BREAKEFIELD XO, FRAEFEL C: HSV-1-based vectors for gene therapy of neurological diseases and brain tumors: part I. HSV-1 structure, replication and pathogenesis. Neoplasia (1999) 1:387–401.
   Google Scholar
• MILLER DA: Retrovirus packaging cells. Hum. Gene Ther. (1990) 1:5–14.
   PubMed Web of Science ®Google Scholar
• RABINOWITZ JE, SAMULSKI J: Adeno-associated virus expression systems for gene transfer. Curc Opin. Biotechnol (1998) 9:470–475.
   PubMed Web of Science ®Google Scholar
• •Excellent summary of AAV expression systems for gene therapy.
   Google Scholar
• GLORIOSO JC, DELUCA NA, FINK DJ: Development and application of herpes simplex virus vectors for human gene therapy. Ann. Rev Microbiol (1995) 49:675–710.
   PubMed Web of Science ®Google Scholar
• HEISTER T, HEID I, ACKERMANN M,FRAEFEL C: Herpes simplex virusType 1/ adeno-associated virus hybrid vectors mediate site-specific integration at the adeno-associated virus preintegration site, AAVS1, on human chromosome 19. Virol (2002) 76:7163–7173.
   Web of Science ®Google Scholar
• WANG Y, CAMP SM, NIWANO M et al.: Herpes simplex virusType l/adeno-associated virus rep(-F) hybrid amplicon vector improves the stability of transgene expression in human cells by site-specific integration.' Virol (2002) 76:7150–7162.
   PubMed Web of Science ®Google Scholar
• MAGUIR-ZEIS KA, BOWERS WJ, FEDEROFF HJ: HSV vector-mediated gene delivery to the central nervous system. Carr. Opin. Mol. Ther. (2001) 3:482–490.
   PubMed Web of Science ®Google Scholar
• YANT SR, EHRHARDT A,MIKKELSEN JG, MEUSE L, PHAM T, KAY MA: Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat. Biotechnol (2002) 20:999–1005.
   PubMed Web of Science ®Google Scholar
• MCKAY R: Reconstituting animals from immortal precursors. CLIFF. Opin. Nem-0Na (1992) 2:582–585.
   PubMedGoogle Scholar
• WHITTEMORE SR, EATON MJ, ONIFER SM: Gene therapy and the use of stem cells for central nervous system regeneration. Adv Neurol (1997) 72:113–119.
   PubMedGoogle Scholar
• TUSZYNSKI MH: Gene therapy for neurodegenerative disorders. Lancet Neurol (2002) 2002:51–57.
   Web of Science ®Google Scholar
• LEVI-MONTALCINI R,HAMBURGER V: Selective growth stimulating effects of mouse sarcoma on the sensory and sympathetic nervous system of the chick embryo.' Exp. Zool (1951) 116:321–362.
   PubMedGoogle Scholar
• ••A landmark description of the discovery ofgrowth factors.
   Google Scholar
• LEVI-MONTALCINI R: The nerve growth factor 35 years later. Science (1987) 237:1154–1162.
   PubMed Web of Science ®Google Scholar
• ••A summary of knowledge regarding NGFactions, as a paper accompanying the award of the Nobel Prize for the discovery of NGE
   Google Scholar
• ANGELETTI RH, HERMODSON MA, BRADSHAW RA: Amino acid sequences of mouse 2.5S nerve growth factor. II. Isolation and characterization of the thermolytic and peptic peptides and the complete covalent structure. Biochemistry (Mose) (1973) 12:100–115.
   Google Scholar
• ULLRICH A, GRAY A, BERMAN C, DULL TJ: Human beta-nerve growth factor gene sequence highly homologous to that of mouse. Nature (1983) 303:821–825.
   PubMed Web of Science ®Google Scholar
• HEFTI F: Nerve growth factor (NGF) promotes survival of septal cholinergic neurons after fimbrial transection. Neurosci. (1986) 6:2155–2162.
   PubMed Web of Science ®Google Scholar
• ••The first report that any growth factorprevents cell death in the adult nervous system.
   Google Scholar
• WILLIAMS LR, VARON S,PETERSON GM et al: Continuous infusion of nerve growth factor prevents basal forebrain neuronal death after fimbria-fornix transection. Proc. Natl. Acad. Sci. USA (1986) 83:9231–9235.
   PubMed Web of Science ®Google Scholar
• KROMER LF: Nerve growth factor treatment after brain injury prevents neuronal death. Science (1987) 235:214–216.
   PubMed Web of Science ®Google Scholar
• FISCHER W, WICTORIN K, BJORKLUND A, WILLIAMS LR, VARON S, GAGE FH: Amelioration of cholinergic neuron atrophy and spatial memory impairment in aged rats by nerve growth factor. Nature (1987) 329:65–68.
   PubMed Web of Science ®Google Scholar
• ••A landmark study that NGF reversesage-related neuronal atrophy and improves memory.
   Google Scholar
• TUSZYNSKI MH, U H-S, AMARAL DG, GAGE FH: Nerve growth factor infusion in primate brain reduces lesion-induced cholinergic neuronal degeneration. Neurosci. (1990) 10:3604–3614.
   PubMed Web of Science ®Google Scholar
• •The first report that growth factors prevent cell degeneration in the primate brain.
   Google Scholar
• KOLIATSOS VE, NAUTA HJ,CLATTERBUCK RE, HOLTZMAN DM, MOBLEY WC, PRICE DL: Mouse nerve growth factor prevents degeneration of axotomized basal forebrain cholinergic neurons in the monkey. Neurosci. (1990) 10:3801–3813.
   PubMed Web of Science ®Google Scholar
• TUSZYNSKI MH, U H-S, GAGE FH: Recombinant human nerve growth factor infusions prevent cholinergic neuronal degeneration in the adult primate brain. Ann. Neurol (1991) 30:625–636.
   PubMed Web of Science ®Google Scholar
• HEFTI F, WEINER WJ: Nerve growth factor and Alzheimer's disease. Ann. Neurol (1986) 20:275–281.
   PubMed Web of Science ®Google Scholar
• PHELPS CH, GAGE FH, GROWDON H et al.: Potential use of nerve growth factor to treat Alzheimer's disease. Neurobiol Aging (1989) 10:205–207.
   PubMed Web of Science ®Google Scholar
• PERRY EK, PERRY RH, BLESSED G, TOMLINSON BE: Necropsy evidence of central cholinergic deficits in senile dementia. Lancet (1977) 2:143.
   Google Scholar
• ••The first report that cholinergicneurons degenerate extensively in AD.
   Google Scholar
• WHITEHOUSE PJ, PRICE DJ, CLARK A, COYLE JT, DELONG M: Alzheimer's disease: evidence for selective loss of cholinergic neurons in the nucleus basalis. Ann. Nemo]. (1981) 10:122–126.
   PubMed Web of Science ®Google Scholar
• PERRY EK, TOMLINSON BE,BLESSED G, BERGMANN K,GIBSON PH, PERRY RH: Correlation of cholinergic abnormalities with senile plaques and mental test scores in senile dementia. Br. Med. (1978) 2:1457–1459.
   PubMed Web of Science ®Google Scholar
• •An important report that loss of cholinergic function parallels cognitive and pathological decline in AD.
   Google Scholar
• WHITEHOUSE PJ, PRICE DL, STRUBLE RG, CLAR AW, COYLE JT, DELONG MR: Alzheimer's disease and senile dementia: loss of neurons in the basal forebrain. Science (1982) 215:1237–1239.
   PubMed Web of Science ®Google Scholar
• •This report, together with the publication below, highlighted the importance of cholinergic system loss in AD.
   Google Scholar
• BARTUS R, DEAN RL, BEER C, LIPPA AS: The cholinergic hypothesis of geriatric memory dysfunction. Science (1982) 217:408–417.
   PubMed Web of Science ®Google Scholar
• •See [33].
   Google Scholar
• CANDY JM, PERRY RH, PERRY EK et al.: Pathological changes in the nucleus basalis of Meynert in Alzheimer's and Parkinson's diseases. .1 Neurosci. (1983) 54:277–289.
   Google Scholar
• MUFSON EJ, CONNER JM,KORDOWER JH: Nerve growth factor in Alzheimer's disease: defective retrograde transport to nucleus basalis. Neuroreport (1995) 6:1063–1066.
   PubMed Web of Science ®Google Scholar
• •Important finding that NGF is deficient in the AD-affected brain.
   Google Scholar
• SCOTT SA, MUFSON EJ, WEINGARTNER JA, SKAU KA, CRUTCHER KA: Nerve growth factor in Alzheimer's disease: increased levels throughout the brain coupled with declines in nucleus basalis. J. Neurosci. (1995) 15:6213–6221.
   PubMed Web of Science ®Google Scholar
• EMMETT CJ, STEWART GR,JOHNSON PM, ASWANI SP, CHAN RL, JAKEMAN LB: Distribution of radioiodinated recombinant human nerve growth factor in primate brain following intracerbroventricular infusion. Exp. Neural. (1996) 140:151–160.
   PubMed Web of Science ®Google Scholar
• ISAACSON LG, SAFFRAN BN, CRUTCHER KA: Intracerebral NGF infusion induces hyperinnervation of cerebral blood vessels. Neurobiol. Aging (1990) 11:51–55.
   PubMed Web of Science ®Google Scholar
• WILLIAMS LR: Hypophagia is induced byintracerebroventricular administration ofnerve growth factor. Exp. Nemo]. (1991) 113:31–37.
   PubMed Web of Science ®Google Scholar
• WINKLER J, RAMIREZ GA, KUHN HG et al.: Reversible Schwann cell hyperplasia and sprouting of sensory and sympathetic neurites after intraventricular administration of nerve growth factor. Ann. Nemo]. (1997) 41:82–93.
   PubMed Web of Science ®Google Scholar
• EMMETT CJ, STEWART GR,JOHNSON RM, ASWANI SP, CHAN RL, JAKEMAN LB: Distribution of radioiodinated recombinant human nerve growth factor in primate brain following intracerebroventricular infusion. Exp. Nemo]. (1996) 140:151–160.
   PubMed Web of Science ®Google Scholar
• ERIKSD OTTER JONHAGEN M, NORDBERG A, AMBERLA K et al.:Intracerebroventricular infusion of nerve growth factor in three patients with Alzheimer's disease. Dement Geriatr Cogn Disord (1998) 9:246–257.
   Google Scholar
• •First clinical trial of NGF in AD, using flawed intraventricular infusions.
   Google Scholar
• ROSENBERG MB, FRIEDMANN T, ROBERTSON RC et al.: Graftinggenetically modified cells to the damaged brain: restorative effects of NGF expression. Science (1988) 242:1575–1578.
   PubMed Web of Science ®Google Scholar
• ••First successful use of gene therapy inthe brain.
   Google Scholar
• CHEN KS, GAGE FH: Somatic gene transfer of NGF to the aged brain: behavioral and morphological amelioration. Neurosci. (1995) 15:2819–2825.
   PubMed Web of Science ®Google Scholar
• TUSZYNSKI MH, ROBERTS J, SENUT MC, U H-S, GAGE FH: Gene therapy in the adult primate brain: intraparenchymal grafts of cells genetically modified to produce nerve growth factor prevent cholinergic neuronal degeneration. Gene Ther. (1996) 3:305–314.
   PubMed Web of Science ®Google Scholar
• EMERICH DW, WINN S, HARPER J, HAMMANG JP, BAETGE EE, KORDOWER JH: Implants of polymer-encapsulated human NGF-secreting cells in the nonhuman primate: rescue and sprouting of degenerating cholinergic basal forebrain neurons. Comp. Nemo]. (1994) 349:148–164.
   PubMed Web of Science ®Google Scholar
• •First report of success of NGF gene delivery in the primate brain.
   Google Scholar
• KORDOWER JH, WINN SR, LIU Y-T et al.: The aged monkey basal forebrain: rescue and sprouting of axotomized basal forebrain neurons after grafts of encapsulated cells secreting human nerve growth factor. Proc. Nati Acad. Sci. (1994) 91:10898–10902.
   PubMed Web of Science ®Google Scholar
• SMITH DE, ROBERTS J, GAGE FH,TUSZYNSKI MH: Age-associated neuronal atrophy occurs in the primate brain and is reversible by growth factor gene therapy. Proc. Nat. Acad. Sci. (1999) 96:10893–10898.
   PubMed Web of Science ®Google Scholar
• •First report that NGF gene therapy reverses spontaneous age-related cholinergic atrophy primates.
   Google Scholar
• DEKKER AJ, LANGDON DJ, GAGE FH, THAL LJ: NGF increases cortical acetylcholine release in rats with lesions of the nucleus basalis. Neuroreport (1991) 2:577–580.
   PubMed Web of Science ®Google Scholar
• MUFSON EJ, CONNER JM,KORDOWER JH: Nerve growth factor inAlzheimer's disease: defective retrograde transport to nucleus basalis. Neuroreport (1995) 6:1063–1066.
   PubMed Web of Science ®Google Scholar
• MANDEL RJ, GAGE FH, CLEVENGER SK, SNYDER RO, LEFF SE: Nerve growth factor expressed in the medial septum following in vivo gene delivery using a recombinant adeno-associated viral vector protects cholinergic neurons from fimbria-fornix lesion-induced degeneration. Exp. Neurol (1999) 155:59–64.
   PubMed Web of Science ®Google Scholar
• KLEIN RL, HIRKO AC, MEYERS CA, GRIMES JR, MUZYCZKA N,MEYER EM: NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged rats. Brain Res. (2000) 875:144–151.
   PubMed Web of Science ®Google Scholar
• TUSZYNSKI MH: Neurotrophic factors. In: CNS Regeneration: Basic Science and Clinical Advances, Tuszynski MH, Kordower JA (Eds), Academic Press, San Diego, USA (1999):109–158.
   Google Scholar
• PHILLIPS HS, HAINS JM, ARAMANI M, LARAMEE GR, JOHNSON SA, WINSLOW JW: BDNF is decreased in the hipocampus of individuals with Alzheimer's disease. Neuron (1991) 7:695–702.
   PubMed Web of Science ®Google Scholar
• GOMEZ-PINILLA F, LEE JW, COTMAN CW: Basic fibroblast growth factor in adult rat brain: cell distribution and response to entorhinal lesion and fimbria-fornix transection. Neurosci. (1992) 12:345–355.
   PubMed Web of Science ®Google Scholar
• PETERSON DA, LUCIDI-PHILLIPI CA, MURPHY DP, RAY J, GAGE FH:Fibroblast growth factor-2 protects entorhinal layer II glutamatergic neurons from axotomy-induced death. Neurosci. (1996) 16:886–898.
   PubMed Web of Science ®Google Scholar
• SPINA MB, HYMAN C, SQUINTO S, LINDSAY RM: Brain-derived neurotrophic factor protects dopaminergic cells from 6-hydroxydopamine toxicity. Ann. NY Acad. Sci. (1992) 648:348–350.
   PubMed Web of Science ®Google Scholar
• UN LF, DOHERTY DH, LILE JD, BEKTESH S, COLLINS F: GDNF: a glial cell-line derived neurotrophic factor for midbrain dopaminergic neurons. Science (1993) 260:1130–1132.
   PubMed Web of Science ®Google Scholar
• FRIM DM, UHLER TA, SHORT MP et al.: Effects of biologically delivered NGF, BDNF and bEGF on striatal excitotoxic lesions. Neuroreport (1993) 4:367–370.
   PubMed Web of Science ®Google Scholar
• HYMAN C, HOFER M, JUHASZ M, YANCOPOULOS GP, SQUINTO SP, LINDSAY RM: Brain derived neurotrophic factor is a neurotrophic factor for dopaminergic neurons of the substantia nigra. Nature (1994) 350:230–232.
   Web of Science ®Google Scholar
• WINKLER C, SAUER H, LEE CS, BJORKLUND A: Short-term GDNF treatment provides long-term rescue of lesioned nigral dopaminergic neurons in a rat model of Parkinson's disease. Neurosci. (1996) 16:7206–7215.
   PubMed Web of Science ®Google Scholar
• KOTZBAUER PT, LAMPE PA, HEUCKEROTH RO et al.: Neurturin, a relative of glial-cell-line derived neurotrophic factor. Nature (1996) 384:467–470.
   PubMed Web of Science ®Google Scholar
• BJORKLUND A, KIRIK D, ROSENBLAD C, GEORGIEVSKA B, LUNDBERG C, MANDEL RJ: Towards a neuroprotective gene therapy for Parkinson's disease: use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in the rat Parkinson model. Brain Res. (2000) 886:82–98.
   PubMed Web of Science ®Google Scholar
• CONNOR B, KOZLOWSKI DA, SCHALLERT T, TILLERSON JL, DAVIDSON BL, BOHN MC: Differential effects of glial cell line-derived neurotrophic factor (GDNF) in the striatum and substantia nigra of the aged Parkinsonian rat. Gene Titer: (1999) 6:1936–1951.
   Google Scholar
• GASH DM, ZHANG Z, OVADIA A et al:Functional recovery in parkinsonian monkeys treated with GDNF. Nature (1996) 380:252–255.
   PubMed Web of Science ®Google Scholar
• •Important finding that GDNF improves symptoms and protects nigral neurons in a primate model of PD.
   Google Scholar
• KORDOWER JH, EMBORG ME, BLOCH J et al.: Neurodegenerationprevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science (2000) 290:767–773.
   PubMed Web of Science ®Google Scholar
• ••Landmark study demonstrating thatGDNF in vivo gene delivery supports nigral neurons in MPTP-lesioned and aged primates, and promotes functional recovery in MPTP-lesioned monkeys.
   Google Scholar
• NUTT JG, BURCHIEL KJ,COMELLA CL et al: Randomized, double-blind trial of glial cell line-derived neurotorphic factor (GDNF) in PD. Neurology (2003) 60:69–73.
   PubMed Web of Science ®Google Scholar
• KIRIK D, ROSENBLAD C,BJORKLUND A, MANDEL R: Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transudction promotes functional regeneration in the lesioned nigrostriatal system. j. Neurosci. (2000) 20:4686–4700.
   PubMed Web of Science ®Google Scholar
• MUFSON EJ, KROIN JS, LIU YT et al.: Intrastriatal and intraventricular infusion of brain-derived neurotrophic factor in the cynomologous monkey: distribution, retrograde transport and co-localization with substantia nigra dopamine-containing neurons. Neuroscience (1996) 71:179–191.
   PubMed Web of Science ®Google Scholar
• WOLFF JA, FISHER LJ, XU L et al: Grafting fibroblasts genetically modified to produce L-dopa in a rat model of Parkinson's disease. Proc. Natl. Acad. Sci. USA (1989) 86:9011–9014.
   PubMed Web of Science ®Google Scholar
• KANG UJ, FISHER LJ, JOH TH, O'MALLEY KL, GAGE FH: Regulation of dopamine production by genetically modified primary fibroblasts. j. Neurosci. (1993) 13:5203–5211.
   PubMed Web of Science ®Google Scholar
• AZZOUZ M, MARTIN-RENDON E, BARBER RD et al.: Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induced sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J. Neurosci. (2002) 22:10302–10312.
   PubMed Web of Science ®Google Scholar
• BANKIEWICZ KS, BRINGAS JR, MCLAUGHLIN W et al.: Application of gene therapy for Parkinson's disease: nonhuman primate experience. Adv. Pharmacol. (1998) 42:801–806.
   PubMedGoogle Scholar
• LUO J, KAPLITT MG,FITZSIMONS HL et al.: Subthalamir GAD gene therapy in a Parkinson's diseaserat model. Science (2002) 298:425–429.
   Google Scholar
• EMERICH DF, LINDNER MD, WINN SR, CHEN EY, FRYDEL BR, KORDOWER JH: Implants of encapsulated human CNTF-producing fibroblasts prevent behavioral deficits and striatal degeneration in a rodent model of Huntington's disease. J. Neurosci. (1996) 16:5168–5181.
   PubMed Web of Science ®Google Scholar
• EMERICH DF, WINN SR,HANTRAYE PM et al.: Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease. Nature (1997) 386:395–399.
   Google Scholar
• •Important report that the growth factor CNTF reverses neuronal degeneration in a primate model of Huntington's disease.
   Google Scholar
• NO AUTHORS LISTED: A double-blindplacebo-controlled trial of subcutaneous recombinant human ciliary neurotrophic factor (rhCNTF) in amyotrophic lateral sclerosis. A-CT GROUP S. Neurology (1996) 46:1244–1249.
   PubMed Web of Science ®Google Scholar
• NO AUTHORS LISTED: A controlled trial of recombinant methionyl human BDNF in ALS: the BDNF Study Group (Phase III). TB GROUP S. Neurology (1999) 52:1427–1433.
   PubMed Web of Science ®Google Scholar
• GRILL R, MURAI K, BLESCH A, GAGE FH, TUSZYNSKI MH: Cellular delivery of neurotrophin-3 promotes corticospinal axonal growth and partial functional recovery after spinal cord injury. Neurosci. (1997) 17:5560–5572.
   PubMed Web of Science ®Google Scholar
• LIU Y, KIM D, HIMES BT et al.:Transplants of fibroblasts genetically modified to express BDNF promote regeneration of adult rat rubrospinal axons and recovery of forelimb function. J. Neurosci. (1999) 19:4370–4387.
   PubMed Web of Science ®Google Scholar
• TUSZYNSKI MH, GRILL R, JONES L, MCKAY HM, BELSCH A: Spontaneous and augmented growth of axons in the primate spinal cord: Effects of local injury and nerve growth factor-secreting cells. J. Comp. Neurol. (2002) 448:88–101.
   Web of Science ®Google Scholar
• LU P, BLESCH A, TUSZYNSKI MH: Neurotrophism without neurotropism: BDNF promotes survival but not growth of lesioned corticospinal neurons. J. Comp. Neurol. (2001) 436:456–470.
   PubMed Web of Science ®Google Scholar
• LEONE P, JANSON CG, BILANIUK L et al.: Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease. Ann. Neurol. (2000) 48:27–38.
   PubMed Web of Science ®Google Scholar
• LIT, DAVIDSON BL: Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer. Proc. Natl. Acad. Sci. USA (1995) 92:7700–7704.
   PubMed Web of Science ®Google Scholar
• •Important study showing benefit of gene therapy in a model of inborn error of metabolism.
   Google Scholar
• STEIN CS, GHODSI A, DERKSEN T, DAVIDSON BL: Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis Type VII Virol (1999) 73:3424–3429.
   Google Scholar
• DALY TM, VOGLER C, LEVY B, HASKINS ME, SANDS MS: Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc. Natl. Acad. Sci. USA (1999) 96:2296–2300.
   PubMed Web of Science ®Google Scholar
• CONSIGLIO A, QUATTRINI A, MARTINO S et al.: Li vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat. Med. (2001) 7:310–316.
   PubMed Web of Science ®Google Scholar
• ALLAMAND V, DONAHUE KM, STRAUB V, DAVISSON RL, DAVIDSON BL, CAMPBELL KP: Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice. Gene The]: (2000) 7:1385–1391.
   PubMed Web of Science ®Google Scholar
• LEARISH R, OHASHI T, ROBBINS PA et al.: Retroviral gene transfer and sustained expression of human arylsulfatase A. Gene Ther. (1996) 3:343–349.
   PubMed Web of Science ®Google Scholar
• LACORAZZA HD, FLAX JD,SNYDER EY, JENDOUBI M: Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells. Nat. Med. (1996) 2:424–429.
   PubMed Web of Science ®Google Scholar
• HAHN CN, DEL PILAR MARTIN M, ZHOU XY, MANN LW, D'AZZO A: Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/ cathepsin A under control of the colony-stimulating factor-1 receptor promoter. Proc Natl. Acad. Sci. USA (1998) 95:14880–14885.
   PubMed Web of Science ®Google Scholar
• TAKENAKA T, MURRAY GJ, QIN G et al.: Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells. Proc. Natl. Acad. Sci. USA (2000) 97:7515–7520.
   PubMed Web of Science ®Google Scholar
• BETZ AL, YANG GY, DAVIDSON BL: Attenuation of stroke size in rats using anadenoviral vector to induce overexpression of interleukin-1 receptor antagonist in brain. Gerd,. Blood Row Metal,. (1995) 15:547–551.
   PubMed Web of Science ®Google Scholar
• O'CONNOR WM, DAVIDSON BL, KAPLITT MG et al: Adenovirus vector-mediated gene transfer into human epileptogenic brain slices: prospects for gene therapy in epilepsy. Exp. Neurol (1997) 148:167–178.
   PubMed Web of Science ®Google Scholar
• SHORT MP, CHOI BC, LEE JK,MALICK A, BREAKEFIELD XO, MARTUZA RL: Gene delivery to glioma cells in rat brain by grafting of a retrovirus packaging cell line. I Neurosci. Res. (1990) 27:427–439.
   PubMed Web of Science ®Google Scholar
• •Description of potential value of gene delivery for nervous system cancer.
   Google Scholar
• BARBA D, HARDIN J, RAY J, GAGE FH: Thymidine kinase-mediated killing of rat brain tumors. I Neurosurg. (1993) 79:729–735.
   PubMed Web of Science ®Google Scholar
• CULVER KW, RAM Z, WALLBRIDGE S,ISHII H, OLDFIELD EH, BLAESE RIVI: Li vivo gene transfer with retroviral vector-producer cells for treatment of experimental brain tumors. Science (1992) 256:1550–1552.
   PubMed Web of Science ®Google Scholar
• NANDA D, DRIESSE MJ, SILLEVIS SMITT PA: Clinical trials of adenoviral-mediated suicide gene therapy of malignant gliomas. Frog. Brain Res. (2001) 132:699–710.
   PubMedGoogle Scholar
• ALAVI JB, ECK SL: Gene therapy for high grade gliomas. Expert Opin. Biol. Ther. (2001) 1:239–252.
   PubMed Web of Science ®Google Scholar
• RAM Z, CULVER KW, OSHIRO EM et al.: Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nat. Med. (1997) 3:1354–1361.
   PubMed Web of Science ®Google Scholar
• SHAND N, WEBER F, MARIANI L et al.: 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 Ther. (1999) 10:2325–2335.
   Google Scholar
• COHEN JL, SARON ME BOYER O etal.: Preservation of graft-versus-infection effects after suicide gene therapy for prevention of graft-versus-host disease. Hum. Gene Tiler. (2000) 11:2473–2481.
   Google Scholar
• RAINOV NG: A Phase III clinical evaluation of herpes simplex virusType 1 thymidine kinase and ganciclovir genetherapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Hum. Gene Ther. (2000) 11:2389–2401.
   PubMed Web of Science ®Google Scholar
• MARKERT JM, PARKER JN,GILLESPIE GY, WHITLEY RJ: Genetically engineered human herpes simplex virus in the treatment of brain tumours. Herpes (2001) 8:17–22.
   PubMedGoogle Scholar
• GOSSEN M, BONIN AL,FREUNDLIEB S, BUJARD H: Inducible gene expression systems for higher eukaryotic cells. Carr. Opin. Biotechnol (1994) 5:516–520.
   PubMedGoogle Scholar
• •Important report indicating possibility of effective, regulated therapeutic gene expression.
   Google Scholar
• BLESCH A, CONNER JM,TUSZYNSKI MH: Modulation of neuronal survival and axonal growth in vivo by tetracycline-regulated neurotrophin expression. Gene Ther. (2001) 8:954–960.
   PubMed Web of Science ®Google Scholar
• FAVRE D, BLOUIN V, PROVOST N et aL: Lack of an immune response against the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated transgene expression in nonhuman primates after intramuscular injection of recombinant adeno-associated virus. Viral. (2002) 76:11605–11611.
   PubMed Web of Science ®Google Scholar
• •Description of potential pitfalls of regulated gene delivery with tetracycline-sensitive system.
   Google Scholar
• SUHR Si GIL EB, SENUT MC, GAGE FH: High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor. Proc. Natl. Acad. Sci. USA (1998) 95:7999–8004.
   PubMed Web of Science ®Google Scholar
• NANDA D, VOGELS R, HAVENGA M, AVEZAAT CJ, BOUT A, SMITT PS: Treatment of malignant gliomas with a replicating adenoviral vector expressing herpes simplex virus-thymidine kinase. Cancer Res. (2001) 61:8743–8750.
   PubMed Web of Science ®Google Scholar