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Genetic Modification of Neural Stem/Progenitor Cells

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References

  • Aberg MA, Ryttsen F, Hellgren G, Lindell K, Rosengren LE, MacLennan AJ, Carlsson B, Orwar O, Eriksson PS (2001) Selective introduction of antisense oligonucleotides into single adult CNS progenitor cells using electroporation demonstrates the requirement of STAT3 activation for CNTF-induced gliogenesis. Mol Cell Neurosci 17: 426–443.

    CAS  PubMed  Google Scholar 

  • Aboody KS, Brown A, Rainov NG, Bower KA, Liu S, Yang W, Small JE, Herrlinger U, Ourednik V, Black PM, Breakefield XO, Snyder EY (2000) Neural stem cells display extensive tropism for pathology in adult brain: evidence from intracranial gliomas. Proc Natl Acad Sci U S A 97: 12846–12851.

    Article  CAS  PubMed  Google Scholar 

  • Ailles LE, Naldini L (2002) HIV-1-derived lentiviral vectors. Curr Top Microbiol Immunol 261: 31–52.

    CAS  PubMed  Google Scholar 

  • Anderson DJ (1999) Lineages and transcription factors in the specification of vertebrate primary sensory neurons. Curr Opin Neurobiol 9: 517–524.

    Article  CAS  PubMed  Google Scholar 

  • Andreansky SS, He B, Gillespie GY, Soroceanu L, Markert J, Chou J, Roizman B, Whitley RJ (1996) The application of genetically engineered herpes simplex viruses to the treatment of experimental brain tumors. Proc Natl Acad Sci U S A 93: 11313–11318.

    Article  CAS  PubMed  Google Scholar 

  • Audouy S, Hoekstra D (2001) Cationic lipid-mediated transfection in vitro and in vivo (review). Mol Membr Biol 18: 129–143.

    CAS  PubMed  Google Scholar 

  • Bartlett JS, Wilcher R, Samulski RJ (2000) Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors. J Virol 74: 2777–2785.

    Article  CAS  PubMed  Google Scholar 

  • Bartlett PF, Reid HH, Bailey KA, Bernard O (1988) Immortalization of mouse neural precursor cells by the c-myc oncogene. Proc Natl Acad Sci U S A 85: 3255–3259.

    CAS  PubMed  Google Scholar 

  • Benedetti S, Pirola B, Polio B, Magrassi L, Bruzzone MG, Rigamonti D, Galli R, Selleri S, Di Meco F, De Fraja C, Vescovi A, Cattaneo E, Finocchiaro G (2000) Gene therapy of experimental brain tumors using neural progenitor cells. Nat Med 6: 447–450.

    CAS  PubMed  Google Scholar 

  • Benraiss A, Chmielnicki E, Lerner K, Roh D, Goldman SA (2001) Adenoviral brain-derived neurotrophic factor induces both neostriatal and olfactory neuronal recruitment from endogenous progenitor cells in the adult forebrain. J Neurosci 21: 6718–6731.

    CAS  PubMed  Google Scholar 

  • Blesch A, Lu P, Tuszynski MH (2002) Neurotrophic factors, gene therapy, and neural stem cells for spinal cord repair. Brain Res Bull 57: 833–838.

    Article  CAS  PubMed  Google Scholar 

  • Bosher JM, Labouesse M (2000) RNA interference: genetic wand and genetic watchdog. Nat Cell Biol 2:E31–E36.

    CAS  PubMed  Google Scholar 

  • Buchet D, Serguera C, Zennou V, Charneau P, Mallet J (2002) Long-term expression of beta-glucuronidase by genetically modified human neural progenitor cells grafted into the mouse central nervous system. Mol Cell Neurosci 19: 389–401.

    Article  CAS  PubMed  Google Scholar 

  • Burton EA, Bai Q, Coins WF, Glorioso JC (2001) Targeting gene expression using HSV vectors. Adv Drug Deliv Rev 53: 155–170.

    Article  CAS  PubMed  Google Scholar 

  • Carpenter MK, Cui X, Hu ZY, Jackson J, Sherman S, Seiger A, Wahlberg LU (1999) In vitro expansion of a multipotent population of human neural progenitor cells. Exp Neurol 158: 265–278.

    Article  CAS  PubMed  Google Scholar 

  • Carter PJ, Samulski RJ (2000) Adeno-associated viral vectors as gene delivery vehicles. Int J Mol Med 6: 17–27.

    CAS  PubMed  Google Scholar 

  • Chambers CB, Peng Y, Nguyen H, Gaiano N, Fishell G, Nye JS (2001) Spatiotemporal selectivity of response to Notchl signals in mammalian forebrain precursors. Development 128: 689–702.

    CAS  PubMed  Google Scholar 

  • Chesnoy S, Huang L (2000) Structure and function of lipid-DNA complexes for gene delivery. Annu Rev Biophys Biomol Struct 29: 27–47.

    Article  CAS  PubMed  Google Scholar 

  • Cho T, Bae JH, Choi HB, Kim SS, McLarnon JG, Suh-Kim H, Kim SU, Min CK (2002) Human neural stem cells: electrophysiological properties of voltage-gated ion channels. Neuroreport 13: 1447–1452.

    CAS  PubMed  Google Scholar 

  • Chow SY, Moul J, Tobias CA, Himes BT, Liu Y, Obrocka M, Hodge L, Tessler A, Fischer I (2000) Characterization and intraspinal grafting of EGF/bFGF-dependent neurospheres derived from embryonic rat spinal cord. Brain Res 874: 87–106.

    Article  CAS  PubMed  Google Scholar 

  • Cornetta K, Morgan RA, Anderson WF (1991) Safety issues related to retroviral-mediated gene transfer in humans. Hum Gene Ther 2: 5–14.

    CAS  PubMed  Google Scholar 

  • Corti O, Horellou P, Colin P, Cattaneo E, Mallet J (1996) Intracerebral tetracycline-dependent regulation of gene expression in grafts of neural precursors. Neuroreport 7: 1655–1659.

    CAS  PubMed  Google Scholar 

  • Corti O, Sabate O, Horellou P, Colin P, Dumas S, Buchet D, Buc-Caron MH, Mallet J (1999) A single adenovirus vector mediates doxycycline-controlled expression of tyrosine hydroxy-lase in brain grafts of human neural progenitors. Nat Biotechnol 17: 349–354.

    CAS  PubMed  Google Scholar 

  • Curran MA, Nolan GP (2002) Nonprimate lentiviral vectors. Curr Top Microbiol Immunol 261: 75–105.

    CAS  PubMed  Google Scholar 

  • Dagle JM, Weeks DL (2001) Oligonucleotide-based strategies to reduce gene expression. Differentiation 69: 75–82.

    Article  CAS  PubMed  Google Scholar 

  • Daly G, Chernajovsky Y (2000) Recent developments in retroviral-mediated gene transduction. Mol Ther 2: 423–434.

    Article  CAS  PubMed  Google Scholar 

  • Danthinne X, Werth E (2000) New tools for the generation of El-and/or E3-substituted adenoviral vectors. Gene Ther 7: 80–87.

    CAS  PubMed  Google Scholar 

  • Davis AA, Temple S (1994) A Self-Renewing Multipotential Stem-Cell in Embryonic Rat Cerebral-Cortex. Nature 372: 263–266.

    CAS  PubMed  Google Scholar 

  • De Smedt SC, Demeester J, Hennink WE (2000) Cationic polymer based gene delivery systems. Pharm Res 17: 113–126.

    PubMed  Google Scholar 

  • Delenda C, Audit M, Danos O (2002) Biosafety issues in lenti vector production. Curr Top Microbiol Immunol 261: 123–141.

    CAS  PubMed  Google Scholar 

  • Duan D, Li Q, Kao AW, Yue Y, Pessin JE, Engelhardt JF (1999) Dynamin is required for recom-binant adeno-associated virus type 2 infection. J Virol 73: 10371–10376.

    CAS  PubMed  Google Scholar 

  • Duan D, Yue Y, Yan Z, Engelhardt JF (2000) A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat Med 6: 595–598.

    CAS  PubMed  Google Scholar 

  • Dunn KJ, Williams BO, Li Y, Pavan WJ (2000) Neural crest-directed gene transfer demonstrates Wntl role in melanocyte expansion and differentiation during mouse development. Proc Natl Acad Sci U S A 97: 10050–10055.

    Article  CAS  PubMed  Google Scholar 

  • Eaton MJ, Whittemore SR (1996) Autocrine BDNF secretion enhances the survival and seroton-ergic differentiation of raphe neuronal precursor cells grafted into the adult rat CNS. Exp Neurol 140: 105–114.

    Article  CAS  PubMed  Google Scholar 

  • Englund U, Bjorklund A, Wictorin K, Lindvall O, Kokaia M (2002a) Grafted neural stem cells develop into functional pyramidal neurons and integrate into host cortical circuitry. Proc Natl Acad Sci U S A 99: 17089–17094.

    Article  CAS  PubMed  Google Scholar 

  • Englund U, Ericson C, Rosenblad C, Mandel RJ, Trono D, Wictorin K, Lundberg C (2000) The use of a recombinant lentiviral vector for ex vivo gene transfer into the rat CNS. Neuroreport 11: 3973–3977.

    CAS  PubMed  Google Scholar 

  • Englund U, Fricker-Gates RA, Lundberg C, Bjorklund A, Wictorin K (2002b) Transplantation of human neural progenitor cells into the neonatal rat brain: extensive migration and differentiation with long-distance axonal projections. Exp Neurol 173: 1–21.

    Article  CAS  PubMed  Google Scholar 

  • Estibeiro P, Godfray J (2001) Antisense as a neuroscience tool and therapeutic agent. Trends Neurosci 24: S56–S62.

    Article  CAS  PubMed  Google Scholar 

  • Evrard C, Borde I, Marin P, Galiana E, Premont J, Gros F, Rouget P (1990) Immortalization of bipotential and plastic glio-neuronal precursor cells. Proc Natl Acad Sci U S A 87: 3062–3066.

    CAS  PubMed  Google Scholar 

  • Falk A, Holmstrom N, Carlen M, Cassidy R, Lundberg C, Frisen J (2002) Gene delivery to adult neural stem cells. Exp Cell Res 279: 34–39.

    Article  CAS  PubMed  Google Scholar 

  • Feigner JH, Kumar R, Sridhar CN, Wheeler CJ, Tsai YJ, Border R, Ramsey P, Martin M, Feigner PL (1994) Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 269: 2550–2561.

    Google Scholar 

  • Ferrari FK, Samulski T, Shenk T, Samulski RJ (1996) Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 70: 3227–3234.

    CAS  PubMed  Google Scholar 

  • Fire A (1999) RNA-triggered gene silencing. Trends Genet 15: 358–363.

    CAS  PubMed  Google Scholar 

  • Fisher KJ, Gao GP, Weitzman MD, DeMatteo R, Burda JF, Wilson JM (1996) Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 70: 520–532.

    CAS  PubMed  Google Scholar 

  • Fisher LJ (1997) Neural precursor cells: applications for the study and repair of the central nervous system. Neurobiol Dis 4: 1–22.

    Article  CAS  PubMed  Google Scholar 

  • Flax JD, Aurora S, Yang C, Simonin C, Wills AM, Billinghurst LL, Jendoubi M, Sidman RL, Wolfe JH, Kim SU, Snyder EY (1998) Engraftable human neural stem cells respond to developmental cues, replace neurons, and express foreign genes. Nat Biotechnol 16: 1033–1039.

    CAS  PubMed  Google Scholar 

  • Foster GA, Stringer BM (1999) Genetic regulatory elements introduced into neural stem and progenitor cell populations. Brain Pathol 9: 547–567.

    CAS  PubMed  Google Scholar 

  • Fox JL (2003) US authorities uphold suspension of SCID gene therapy. Nat Biotechnol 21: 217.

    CAS  PubMed  Google Scholar 

  • Fraefel C, Jacoby DR, Breakefield XO (2000) Herpes simplex virus type 1-based amplicon vector systems. Adv Virus Res 55: 425–451.

    CAS  PubMed  Google Scholar 

  • Frederiksen K, Jat PS, Valtz N, Levy D, McKay R (1988) Immortalization of precursor cells from the mammalian CNS. Neuron 1: 439–448.

    Article  CAS  PubMed  Google Scholar 

  • Friedmann T, Yee JK (1995) Pseudotyped retroviral vectors for studies of human gene therapy. NatMed 1:275–277.

    CAS  Google Scholar 

  • Fults D, Pedone C, Dai C, Holland EC (2002) MYC expression promotes the proliferation of neural progenitor cells in culture and in vivo. Neoplasia 4: 32–39.

    Article  CAS  PubMed  Google Scholar 

  • Gage FH, Coates PW, Palmer TD, Kuhn HG, Fisher LJ, Suhonen JO, Peterson DA, Suhr ST, Ray J (1995) Survival and differentiation of adult neuronal progenitor cells transplanted to the adult brain. Proc Natl Acad Sri U S A 92: 11879–11883.

    CAS  Google Scholar 

  • Galimi F, Verma IM (2002) Opportunities for the use of lentiviral vectors in human gene therapy. Curr Top Microbiol Immunol 261: 245–254.

    CAS  PubMed  Google Scholar 

  • Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM (2002) Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854–11859.

    CAS  PubMed  Google Scholar 

  • Giannoukakis N, Thomson A, Robbins P (1999) Gene therapy in transplantation. Gene Ther 6: 1499–1511.

    CAS  PubMed  Google Scholar 

  • Grimm D, Kern A, Rittner K, Kleinschmidt JA (1998) Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther 9: 2745–2760.

    CAS  PubMed  Google Scholar 

  • Guillot C, Le Mauff B, Cuturi MC, Anegon I (2000) Gene therapy in transplantation in the year 2000: moving towards clinical applications? Gene Ther 7: 14–19.

    Article  CAS  PubMed  Google Scholar 

  • Hammond SM, Caudy AA, Hannon GJ (2001) Post-transcriptional gene silencing by doublestranded RNA. Nat Rev Genet 2: 110–119.

    Article  CAS  PubMed  Google Scholar 

  • Hannon GJ (2002) RNA interference. Nature 418: 244–251.

    Article  CAS  PubMed  Google Scholar 

  • Herrlinger U, Woiciechowski C, Sena-Esteves M, Aboody KS, Jacobs AH, Rainov NG, Snyder EY, Breakefield XO (2000) Neural precursor cells for delivery of replication-conditional HSV-1 vectors to intracerebral gliomas. Mol Ther 1: 347–357.

    Article  CAS  PubMed  Google Scholar 

  • Hitt MM, Graham FL (2000) Adenovirus vectors for human gene therapy. Adv Virus Res 55: 479–505.

    CAS  PubMed  Google Scholar 

  • Holt CE, Bertsch TW, Ellis HM, Harris WA (1988) Cellular determination in the Xenopus retina is independent of lineage and birth date. Neuron 1: 15–26.

    Article  CAS  PubMed  Google Scholar 

  • Hoshimaru M, Ray J, Sah DW, Gage FH (1996) Differentiation of the immortalized adult neuronal progenitor cell line HC2S2 into neurons by regulatable suppression of the v-myc oncogene. Proc Natl Acad Sci U S A 93: 1518–1523.

    Article  CAS  PubMed  Google Scholar 

  • Hubener M, Gotz M, Klostermann S, Bolz J (1995) Guidance of thalamocortical axons by growth-promoting molecules in developing rat cerebral cortex. Eur J Neurosci 7: 1963–1972.

    CAS  PubMed  Google Scholar 

  • Hughes SM, Moussavi-Harami F, Sauter SL, Davidson BL (2002) Viral-mediated gene transfer to mouse primary neural progenitor cells. Mol Ther 5: 16–24.

    Article  CAS  PubMed  Google Scholar 

  • Jaaskelainen I, Urtti A (2002) Cell membranes as barriers for the use of antisense therapeutic agents. Mini Rev Med Chem 2: 307–318.

    Article  CAS  PubMed  Google Scholar 

  • Jacobs A, Breakefield XO, Fraefel C (1999) HSV-1-based vectors for gene therapy of neurological diseases and brain tumors: part II. Vector systems and applications. Neoplasia 1: 402–416.

    CAS  PubMed  Google Scholar 

  • Johansson CB, Svensson M, Wallstedt L, Janson AM, Frisen J (1999) Neural stem cells in the adult human brain. Exp Cell Res 253: 733–736.

    Article  CAS  PubMed  Google Scholar 

  • Johe KK, Hazel TG, Muller T, Dugich-Djordjevic MM, McKay RD (1996) Single factors direct the differentiation of stem cells from the fetal and adult central nervous system. Genes Dev 10: 3129–3140.

    CAS  PubMed  Google Scholar 

  • Jungbluth S, Bell E, Lumsden A (1999) Specification of distinct motor neuron identities by the singular activities of individual Hox genes. Development 126: 2751–2758.

    CAS  PubMed  Google Scholar 

  • Kafri T (2001) Lentivirus vectors: difficulties and hopes before clinical trials. Curr Opin Mol Ther 3: 316–326.

    CAS  PubMed  Google Scholar 

  • Kalyani A, Hobson K, Rao MS (1997) Neuroepithelial stem cells from the embryonic spinal cord: Isolation, characterization, and clonal analysis. Dev Biol 186: 202–223.

    Article  CAS  PubMed  Google Scholar 

  • Kim SH, Kim S, Robbins PD (2000) Retroviral vectors. Adv Virus Res 55: 545–563.

    CAS  PubMed  Google Scholar 

  • Kim YC, Shim JW, Oh YJ, Son H, Lee YS, Lee SH (2002) Co-transfection with cDNA encoding the Bel family of anti-apoptotic proteins improves the efficiency of transfection in primary fetal neural stem cells. J Neurosci Methods 117: 153–158.

    Article  CAS  PubMed  Google Scholar 

  • Kotin RM, Siniscalco M, Samulski RJ, Zhu XD, Hunter L, Laughlin CA, McLaughlin S, Muzyczka N, Rocchi M, Berns KI (1990) Site-specific integration by adeno-associated virus. Proc Natl Acad Sri U S A 87: 2211–2215.

    CAS  Google Scholar 

  • Lachmann RH, Efstathiou S (1999) Gene transfer with herpes simplex vectors. Curr Opin Mol Ther 1: 622–632.

    CAS  PubMed  Google Scholar 

  • Lacorazza HD, Flax JD, Snyder EY, Jendoubi M (1996) 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 2: 424–429.

    Article  CAS  PubMed  Google Scholar 

  • Leber SM, Breedlove SM, Sanes JR (1990) Lineage, arrangement, and death of clonally related motoneurons in chick spinal cord. J Neurosci 10: 2451–2462.

    CAS  PubMed  Google Scholar 

  • Lemkine GF, Mantero S, Migne C, Raji A, Goula D, Normandie P, Levi G, Demeneix BA (2002) Preferential transfection of adult mouse neural stem cells and their immediate progeny in vivo with polyethylenimine. Mol Cell Neurosci 19: 165–174.

    Article  CAS  PubMed  Google Scholar 

  • Lever AM (2000) Lentiviral vectors: progress and potential. Curr Opin Mol Ther 2: 488–496.

    CAS  PubMed  Google Scholar 

  • Liu Y, Himes BT, Solowska J, Moul J, Chow SY, Park KI, Tessler A, Murray M, Snyder EY, Fischer I (1999) Intraspinal delivery of neurotrophin-3 using neural stem cells genetically modified by recombinant retrovirus. Exp Neurol 158: 9–26.

    CAS  PubMed  Google Scholar 

  • Lundberg C, Martinez-Serrano A, Cattaneo E, McKay RD, Bjorklund A (1997) Survival, integration, and differentiation of neural stem cell lines after transplantation to the adult rat striaturn. Exp Neurol 145: 342–360.

    Article  CAS  PubMed  Google Scholar 

  • Luskin MB (1993) Restricted proliferation and migration of postnatally generated neurons derived from the forebrain subventricular zone. Neuron 11: 173–189.

    Article  CAS  PubMed  Google Scholar 

  • Luskin MB, Pearlman AL, Sanes JR (1988) Cell lineage in the cerebral cortex of the mouse studied in vivo and in vitro with a recombinant retrovirus. Neuron 1: 635–647.

    Article  CAS  PubMed  Google Scholar 

  • Martínez-Serrano A, Bjorklund A (1996) Protection of the neostriatum against excitotoxic damage by neurotrophin-producing, genetically modified neural stem cells. J Neurosci 16: 4604–4616.

    PubMed  Google Scholar 

  • Martínez-Serrano A, Bjorklund A (1997) Immortalized neural progenitor cells for CNS gene transfer and repair. Trends Neurosci 20: 530–538.

    Article  PubMed  Google Scholar 

  • Martínez-Serrano A, Bjorklund A (1998) Ex vivo nerve growth factor gene transfer to the basal forebrain in presymptomatic middle-aged rats prevents the development of cholinergic neuron atrophy and cognitive impairment during aging. Proc Natl Acad Sci U S A 95: 1858–1863.

    PubMed  Google Scholar 

  • Martínez-Serrano A, Fischer W, Bjorklund A (1995a) Reversal of age-dependent cognitive impairments and cholinergic neuron atrophy by NGF-secreting neural progenitors grafted to the basal forebrain. Neuron 15: 473–484.

    PubMed  Google Scholar 

  • Martínez-Serrano A, Lundberg C, Horellou P, Fischer W, Bentlage C, Campbell K, McKay RD, Mallet J, Bjorklund A (1995b) CNS-derived neural progenitor cells for gene transfer of nerve growth factor to the adult rat brain: complete rescue of axotomized cholinergic neurons after transplantation into the septum. J Neurosci 15: 5668–5680.

    PubMed  Google Scholar 

  • Martínez-Serrano A, Rubio FJ, Navarro B, Bueno C, Villa A(2001) Human neural stem and progenitor cells: in vitro and in vivo properties, and potential for gene therapy and cell replacement in the CNS. Curr Gene Ther 1: 279–299.

    PubMed  Google Scholar 

  • Martínez-Serrano A, Villa A, Navarro B, Rubio FJ, Bueno C (2000) Human neural progenitor cells: better blue than green? Nat Med 6: 483–484.

    Article  PubMed  Google Scholar 

  • McManus MT, Sharp PA (2002) Gene silencing in mammals by small interfering RNAs. Nat Rev Genet 3: 737–747.

    Article  CAS  PubMed  Google Scholar 

  • Merdan T, Kopecek J, Kissel T (2002) Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv Drug Deliv Rev 54: 715–758.

    Article  CAS  PubMed  Google Scholar 

  • Meyers C, Mane M, Kokorina N, Alam S, Hermonat PL (2000) Ubiquitous human adeno-associated virus type 2 autonomously replicates in differentiating keratinocytes of a normal skin model. Virology 272: 338–346.

    Article  CAS  PubMed  Google Scholar 

  • Mizumoto H, Mizumoto K, Whiteley SJ, Shatos M, Klassen H, Young MJ (2001) Transplantation of human neural progenitor cells to the vitreous cavity of the Royal College of Surgeons rat. Cell Transplant 10: 223–233.

    CAS  PubMed  Google Scholar 

  • Monahan PE, Samulski RJ (2000) Adeno-associated virus vectors for gene therapy: more pros than cons? Mol Med Today 6: 433–440.

    CAS  PubMed  Google Scholar 

  • Morgan JC, Majors JE, Galileo DS (2000) Wild-type and mutant forms of v-src differentially alter neuronal migration and differentiation in vivo. J Neurosci Res 59: 226–237.

    Article  CAS  PubMed  Google Scholar 

  • Morshead CM, Craig CG, van der KD (1998) In vivo clonal analyses reveal the properties of endogenous neural stem cell proliferation in the adult mammalian forebrain. Development 125:2251–2261.

    CAS  PubMed  Google Scholar 

  • Muzyczka N (1992) Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol 158: 97–129.

    CAS  PubMed  Google Scholar 

  • Muzyczka N, Berns KI (2001) Parvoviridae: The Viruses and Their Replication. In: Fundamental Virology (Knipe DM, Howley PM, Griffin D., eds), pp 1089–1121. Philadelphia: Lippincott Williams & Wilkins.

    Google Scholar 

  • Naldini L (1998) Lentiviruses as gene transfer agents for delivery to non-dividing cells. Curr Opin Biotechnol 9: 457–463.

    Article  CAS  PubMed  Google Scholar 

  • Naldini L, Blomer U, Gage FH, Trono D, Verma IM (1996a) Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci U S A 93: 11382–11388.

    Article  CAS  PubMed  Google Scholar 

  • Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D (1996b) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263–267.

    CAS  PubMed  Google Scholar 

  • Naldini L, Verma IM (2000) Lentiviral vectors. Adv Virus Res 55: 599–609.

    CAS  PubMed  Google Scholar 

  • Nasz I, Adam E (2001) Recombinant adenovirus vectors for gene therapy and clinical trials. Acta Microbiol Immunol Hung 48: 323–348.

    CAS  PubMed  Google Scholar 

  • NIH Report (2002) Assessment of adenoviral vector safety and toxicity: report of the National Institutes of Health Recombinant DNA Advisory Committee. Hum Gene Ther 13: 3–13.

    Google Scholar 

  • Opalinska JB, Gewirtz AM (2002) Nucleic-acid therapeutics: basic principles and recent applications. Nat Rev Drug Discov 1: 503–514.

    Article  CAS  PubMed  Google Scholar 

  • Ostenfeld T, Tai YT, Martin P, Deglon N, Aebischer P, Svendsen CN (2002) Neurospheres modified to produce glial cell line-derived neurotrophic factor increase the survival of transplanted dopamine neurons. J Neurosci Res 69: 955–965.

    Article  CAS  PubMed  Google Scholar 

  • Ourednik V, Ourednik J, Park KI, Snyder EY (1999) Neural stem cells — a versatile tool for cell replacement and gene therapy in the central nervous system. Clin Genet 56: 267–278.

    Article  CAS  PubMed  Google Scholar 

  • Owens GC, Mistry S, Edelman GM, Crossin KL (2002) Efficient marking of neural stem cellderived neurons with a modified murine embryonic stem cell virus, MESV2. Gene Ther 9: 1044–1048.

    Article  CAS  PubMed  Google Scholar 

  • Owens RA (2002) Second generation adeno-associated virus type 2-based gene therapy systems with the potential for preferential integration into AAVS1. Curr Gene Ther 2: 145–159.

    CAS  PubMed  Google Scholar 

  • Palmer TD, Schwartz PH, Taupin P, Kaspar B, Stein SA, Gage FH (2001) Cell culture. Progenitor cells from human brain after death. Nature 411: 42–43.

    Article  CAS  PubMed  Google Scholar 

  • Palu G, Parolin C, Takeuchi Y, Pizzato M (2000) Progress with retroviral gene vectors. Rev Med Virol 10: 185–202.

    CAS  PubMed  Google Scholar 

  • Pannell D, Ellis J (2001) Silencing of gene expression: implications for design of retrovirus vectors. Rev Med Virol 11: 205–217.

    Article  CAS  PubMed  Google Scholar 

  • Park KI, Ourednik J, Ourednik V, Taylor RM, Aboody KS, Auguste KI, Lachyankar MB, Redmond DE, Snyder EY (2002) Global gene and cell replacement strategies via stem cells. Gene Ther 9: 613–624.

    Article  CAS  PubMed  Google Scholar 

  • Parolin C, Dorfman T, Palu G, Gottlinger H, Sodroski J (1994) Analysis in human immunodeficiency virus type 1 vectors of cis-acting sequences that affect gene transfer into human lymphocytes. J Virol 68: 3888–3895.

    CAS  PubMed  Google Scholar 

  • Pedroso de Lima MC, Simoes S, Pires P, Faneca H, Duzgunes N (2001) Cationic lipid-DNA complexes in gene delivery: from biophysics to biological applications. Adv Drug Deliv Rev 47: 277–294.

    Article  CAS  PubMed  Google Scholar 

  • Pluchino S, Quattrini A, Brambilla E, Gritti A, Salani G, Dina G, Galli R, Del Cairo U, Amadio S, Bergami A, Furlan R, Comi G, Vescovi AL, Martino G (2003) Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. Nature 422: 688–694.

    Article  CAS  PubMed  Google Scholar 

  • Qing K, Mah C, Hansen J, Zhou S, Dwarki V, Srivastava A (1999) Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med 5: 71–77.

    Article  CAS  PubMed  Google Scholar 

  • Rabinowitz JE, Samulski RJ (2000) Building a better vector: the manipulation of AAV virions. Virology 278: 301–308.

    Article  CAS  PubMed  Google Scholar 

  • Redies C, Lendahl U, McKay RD (1991) Differentiation and heterogeneity in T-antigen immortalized precursor cell lines from mouse cerebellum. J Neurosci Res 30: 601–615.

    Article  CAS  PubMed  Google Scholar 

  • Reynolds BA, Tetzlaff W, Weiss S (1992) A multipotent EGF-responsive striatal embryonic progenitor cell produces neurons and astrocytes. J Neurosci 12: 4565–4574.

    CAS  PubMed  Google Scholar 

  • Roizman B, Knipe DM (2003) Herpes Simplex Viruses and Their Replication. In: Fundamental Virology (Knipe DM, Howley PM, Griffin D., eds), pp 1123–1184. Philadelphia: Lippincott Williams & Wilkins.

    Google Scholar 

  • Rosenqvist N, Hard Af SC, Samuelsson C, Johansen J, Lundberg C (2002) Activation of silenced transgene expression in neural precursor cell lines by inhibitors of histone deacetylation. J Gene Med 4: 248–257.

    Article  PubMed  Google Scholar 

  • Roy NS, Wang S, Jiang L, Kang J, Benraiss A, Harrison-Restelli C, Fraser RA, Couldwell WT, Kawaguchi A, Okano H, Nedergaard M, Goldman SA (2000) In vitro neurogenesis by progenitor cells isolated from the adult human hippocampus. Nat Med 6: 271–277.

    CAS  PubMed  Google Scholar 

  • Russell WC (2000) Update on adenovirus and its vectors. J Gen Virol 81: 2573–2604.

    CAS  PubMed  Google Scholar 

  • Ryder EF, Snyder EY, Cepko CL (1990) Establishment and characterization of multipotent neural cell lines using retrovirus vector-mediated oncogene transfer. J Neurobiol 21: 356–375.

    Article  CAS  PubMed  Google Scholar 

  • Sabate O, Horellou P, Vigne E, Colin P, Perricaudet M, Buc-Caron MH, Mallet J (1995) Transplantation to the rat brain of human neural progenitors that were genetically modified using adenoviruses. Nat Genet 9: 256–260.

    CAS  PubMed  Google Scholar 

  • Sakakibara S, Nakamura Y, Yoshida T, Shibata S, Koike M, Takano H, Ueda S, Uchiyama Y, Noda T, Okano H (2002) RNA-binding protein Musashi family: roles for CNS stem cells and a subpopulation of ependymal cells revealed by targeted disruption and antisense ablation. Proc Natl Acad Sci U S A 99: 15194–15199.

    Article  CAS  PubMed  Google Scholar 

  • Samulski RJ, Zhu X, Xiao X, Brook JD, Housman DE, Epstein N, Hunter LA (1991) Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 10: 3941–3950.

    CAS  PubMed  Google Scholar 

  • Sanlioglu S, Monick MM, Luleci G, Hunninghake GW, Engelhardt JF (2001) Rate limiting steps of AAV transduction and implications for human gene therapy. Curr Gene Ther 1: 137–147.

    CAS  PubMed  Google Scholar 

  • Shihabuddin LS, Ray J, Gage FH (1999) Stem cell technology for basic science and clinical applications. Arch Neurol 56: 29–32.

    Article  CAS  PubMed  Google Scholar 

  • Simonato M, Manservigi R, Marconi P, Glorioso J (2000) Gene transfer into neurones for the molecular analysis of behaviour: focus on herpes simplex vectors. Trends Neurosci 23: 183190.

    Article  Google Scholar 

  • Smith-Arica JR, Bartlett JS (2001) Gene therapy: recombinant adeno-associated virus vectors. Curr Cardiol Rep 3: 43–49.

    CAS  PubMed  Google Scholar 

  • Snyder EY, Deitcher DL, Walsh C, Arnold-Aldea S, Hartwieg EA, Cepko CL (1992) Multipotent neural cell lines can engraft and participate in development of mouse cerebellum. Cell 68: 33–51.

    Article  CAS  PubMed  Google Scholar 

  • Snyder EY, Taylor RM, Wolfe JH (1995) Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 374: 367–370.

    Article  CAS  PubMed  Google Scholar 

  • Song H, Stevens CF, Gage FH (2002) Astroglia induce neurogenesis from adult neural stem cells. Nature 417: 39–44.

    Article  CAS  PubMed  Google Scholar 

  • Stemple DL, Anderson DJ (1992) Isolation of A Stem-Cell for Neurons and Glia from the Mammalian Neural Crest. Cell 71: 973–985.

    Article  CAS  PubMed  Google Scholar 

  • Stevenson M (2002) Molecular biology of lenti virus-mediated gene transfer. Curr Top Microbiol Immunol 261: 1–30.

    CAS  PubMed  Google Scholar 

  • Summerford C, Bartlett JS, Samulski RJ (1999) AlphaVbeta5 integrin: a co-receptor for adenoassociated virus type 2 infection. Nat Med 5: 78–82.

    CAS  PubMed  Google Scholar 

  • Suzuki A, Obi K, Urabe T, Hayakawa H, Yamada M, Kaneko S, Onodera M, Mizuno Y, Mochizuki H (2002) Feasibility of ex vivo gene therapy for neurological disorders using the new retroviral vector GCDNsap packaged in the vesicular stomatitis virus G protein. J Neurochem 82: 953–960.

    Article  CAS  PubMed  Google Scholar 

  • Svendsen CN, ter Borg MG, Armstrong RJE, Rosser AE, Chandran S, Ostenfeld T, Caldwell MA (1998) A new method for the rapid and long term growth of human neural precursor cells. J Neurosci Methods 85: 141–152.

    Article  CAS  PubMed  Google Scholar 

  • Temple S (1989) Division and Differentiation of Isolated Cns Blast Cells in Microculture. Nature 340: 471–473.

    Article  CAS  PubMed  Google Scholar 

  • Torchiana E, Lulli L, Cattaneo E, Invernizzi F, Orefice R, Bertagnolio B, Di Donate S, Finocchiaro G (1998) Retroviral-mediated transfer of the galactocerebrosidase gene in neural progenitor cells. Neuroreport 9: 3823–3827.

    CAS  PubMed  Google Scholar 

  • Turner DL, Cepko CL (1987) A common progenitor for neurons and glia persists in rat retina late in development. Nature 328: 131–136.

    Article  CAS  PubMed  Google Scholar 

  • Tuschl T (2002) Expanding small RNA interference. Nat Biotechnol 20: 446–448.

    Article  CAS  PubMed  Google Scholar 

  • Uchida K, Toya S (1996) Grafting of genetically manipulated cells into adult brain: toward graftgene therapy. Keio J Med 45: 81–89.

    CAS  PubMed  Google Scholar 

  • Uchida N, Buck DW, He D, Reitsma MJ, Masek M, Phan TV, Tsukamoto AS, Gage FH, Weissman IL (2000) Direct isolation of human central nervous system stem cells. Proc Natl Acad Sci U S A 97: 14720–14725.

    Article  CAS  PubMed  Google Scholar 

  • van Praag H, Schinder AF, Christie BR, Toni N, Palmer TD, Gage FH (2002) Functional neurogenesis in the adult hippocampus. Nature 415: 1030–1034.

    PubMed  Google Scholar 

  • Vescovi AL, Snyder EY (1999) Establishment and properties of neural stem cell clones: plasticity in vitro and in vivo. Brain Pathol 9: 569–598.

    CAS  PubMed  Google Scholar 

  • Vicario I, Schimmang T (2003) Transfer of FGF-2 viaHSV-1-based amplicon vectors promotes efficient formation of neurons from embryonic stem cells. J Neurosci Methods 123: 55–60.

    Article  CAS  PubMed  Google Scholar 

  • Villa A, Snyder EY, Vescovi A, Martinez-Serrano A (2000) Establishment and properties of a growth factor-dependent, perpetual neural stem cell line from the human CNS. Exp Neurol 161: 67–84.

    Article  CAS  PubMed  Google Scholar 

  • Wagner J, Akerud P, Castro DS, Holm PC, Canals JM, Snyder EY, Perlmann T, Arenas E (1999) Induction of a midbrain dopaminergic phenotype in Nurrl-overexpressing neural stem cells by type 1 astrocytes. Nat Biotechnol 17: 653–659.

    CAS  PubMed  Google Scholar 

  • Wang S, Roy NS, Benraiss A, Goldman SA (2000) Promoter-based isolation and fluorescenceactivated sorting of mitotic neuronal progenitor cells from the adult mammalian ependymal/subependymal zone. Dev Neurosci 22: 167–176.

    Article  PubMed  Google Scholar 

  • Wang S, Wu H, Jiang J, Delohery TM, Isdell F, Goldman SA (1998) Isolation of neuronal precursors by sorting embryonic forebrain transfected with GFP regulated by the T alpha 1 tubulin promoter. Nat Biotechnol 16: 196–201.

    Article  CAS  PubMed  Google Scholar 

  • Wetts R, Fraser SE (1988) Multipotent precursors can give rise to all major cell types of the frog retina. Science 239: 1142–1145.

    CAS  PubMed  Google Scholar 

  • Whittemore SR, White LA (1993) Target regulation of neuronal differentiation in a temperaturesensitive cell line derived from medullary raphe. Brain Res 615: 27–40.

    Article  CAS  PubMed  Google Scholar 

  • Williams BP, Price J (1995) Evidence for multiple precursor cell types in the embryonic rat cerebral cortex. Neuron 14: 1181–1188.

    Article  CAS  PubMed  Google Scholar 

  • Wu P, Phillips MI, Bui J, Terwilliger EF (1998) Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets. J Virol 72: 5919–5926.

    CAS  PubMed  Google Scholar 

  • Wu P, Tarasenko YI, Gu Y, Huang LY, Coggeshall RE, Yu Y (2002a) Region-specific generation of cholinergic neurons from fetal human neural stem cells grafted in adult rat. Nat Neurosci 5: 1271–1278.

    Article  CAS  PubMed  Google Scholar 

  • Wu P, Ye Y, Svendsen CN (2002b) Transduction of human neural progenitor cells using recombinant adeno-associated viral vectors. Gene Ther 9: 245–255.

    Article  CAS  PubMed  Google Scholar 

  • Xiao X, Li J, Samulski RJ (1998) Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 72: 2224–2232.

    CAS  PubMed  Google Scholar 

  • Yakobson B, Hrynko TA, Peak MJ, Winocour E (1989) Replication of adeno-associated virus in cells irradiated with UV light at 254 nm. J Virol 63: 1023–1030.

    CAS  PubMed  Google Scholar 

  • Yalkinoglu AO, Zentgraf H, Hubscher U (1991) Origin of adeno-associated virus DNA replication is a target of carcinogen-inducible DNA amplification. J Virol 65: 3175–3184.

    CAS  PubMed  Google Scholar 

  • Yang CC, Xiao X, Zhu X, Ansardi DC, Epstein ND, Frey MR, Matera AG, Samulski RJ (1997) Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J Virol 71: 9231–9247.

    CAS  PubMed  Google Scholar 

  • Yoon SO, Lois C, Alvirez M, Alvarez-Buylla A, Falck-Pedersen E, Chao MV (1996) Adenovirus-mediated gene delivery into neuronal precursors of the adult mouse brain. Proc Natl Acad Sci U S A 93: 11974–11979.

    CAS  PubMed  Google Scholar 

  • Young MJ, Ray J, Whiteley SJ, Klassen H, Gage FH (2000) Neuronal differentiation and morphological integration of hippocampal progenitor cells transplanted to the retina of immature and mature dystrophic rats. Mol Cell Neurosci 16: 197–205.

    Article  CAS  PubMed  Google Scholar 

  • Zamore PD (2001) RNA interference: listening to the sound of silence. Nat Struct Biol 8: 746750.

    Article  Google Scholar 

  • Zennou V, Serguera C, Sarkis C, Colin P, Perret E, Mallet J, Charneau P (2001) The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain. Nat Biotechnol 19: 446–450.

    Article  CAS  PubMed  Google Scholar 

  • Zhao N, Liu DP, Liang CC (2001) Hot topics in adeno-associated virus as a gene transfer vector. Mol Biotechnol 19:229–237.

    Article  CAS  PubMed  Google Scholar 

  • Zlokovic BV, Apuzzo ML (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: 805–812.

    CAS  PubMed  Google Scholar 

  • Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I, Potter M, Chesnut K, Summerford C, Samulski RJ, Muzyczka N (1999) Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 6: 973–985.

    Article  CAS  PubMed  Google Scholar 

  • Zufferey R, Donello JE, Trono D, Hope TJ (1999) Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 73: 2886–2892.

    CAS  PubMed  Google Scholar 

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Wu, P., Xiao, W. (2003). Genetic Modification of Neural Stem/Progenitor Cells. In: Bottenstein, J.E. (eds) Neural Stem Cells. Springer, Boston, MA. https://doi.org/10.1007/0-306-48356-4_14

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