Neuronal precursor-restricted transduction via in utero CNS gene delivery of a novel bipartite HSV amplicon/transposase hybrid vector

Molecular Therapy

Volumn 13, Issue 3, 2006, Pages 580-588

  Bowers, William J ^a   Mastrangelo, Michael A ^a   Howard, Darlene F ^a   Southerland, Hilary A ^a   Maguire Zeiss, Kathleen A ^a   Federoff, Howard J ^a  

^a UNIVERSITY OF ROCHESTER SCHOOL OF MEDICINE AND DENTISTRY   (United States)

Author keywords

Gene transfer; HSV amplicon; In utero; Neuroprogenitor cell; Sleeping Beauty; Transposon

Indexed keywords

BETA GALACTOSIDASE; GENE PRODUCT; NEOMYCIN; TRANSPOSASE; VIRUS FUSION PROTEIN;

AMPLICON; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CATALYSIS; CELL LINEAGE; CELL METABOLISM; CELL NUCLEUS; CELL SURVIVAL; CELL TYPE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DNA MODIFICATION; EMBRYO; EXPRESSION VECTOR; FEMALE; FUSION GENE; GENE EXPRESSION; GENE TARGETING; GENETIC DISORDER; GENETIC TRANSDUCTION; HERPES SIMPLEX VIRUS; IMMEDIATE EARLY GENE; LONG TERMINAL REPEAT; MITOSIS; MOUSE; NERVOUS SYSTEM DEVELOPMENT; NEURAL STEM CELL; NONHUMAN; NUCLEOTIDE SEQUENCE; PROMOTER REGION; ROUS SARCOMA ONCOVIRUS; TRANSGENE; TRANSPOSON; VIRUS GENE;

ANIMALS; BETA-GALACTOSIDASE; BRAIN; CELL LINE; CRICETINAE; FEMALE; FETUS; GENETIC VECTORS; MICE; MICE, INBRED C57BL; NEURONS; NIH 3T3 CELLS; PREGNANCY; SIMPLEXVIRUS; STEM CELL TRANSPLANTATION; TRANSDUCTION, GENETIC; TRANSPOSASES;

ANIMALIA; ROUS SARCOMA VIRUS; SIMPLEXVIRUS;

EID: 32944457236     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.11.011     Document Type: Article

References (33)

1. F.H. Gage J. Ray L.J. Fisher Isolation, characterization, and use of stem cells from the CNS Annu. Rev. Neurosci. 18 1995 159-192
2. R. McKay Stem cells in the central nervous system Science 276 1997 66-71
3. A. Oehmig C. Fraefel X.O. Breakefield Update on herpesvirus amplicon vectors Mol. Ther. 10 2004 630-643
4. H.J. Federoff M.D. Geschwind A.I. Geller J.A. Kessler Expression of nerve growth factor in vivo, from a defective HSV-1 vector prevents effects of axotomy on sympathetic ganglia Proc. Natl. Acad. Sci. USA 89 1992 1636-1640
5. A.I. Geller X.O. Breakefield A defective HSV-1 vector expresses Escherichia coli ́galactosidase in cultured peripheral neurons Science 241 1988 1667-1669
6. N.D. Stow E. McMonagle Propagation of foreign DNA sequences linked to a herpes simplex virus origin of replication Y. Gluzman Eucaryotic Viral Vectors 1982 Cold Spring Harbor Laboratory Press Cold Spring Harbor, NY 199-204
7. R.R. Spaete N. Frenkel The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning-amplifying vector Cell 30 1982 305-310
8. M.G. Kaplitt A.D. Kwong S.P. Kleopoulos C.V. Mobbs S.D. Rabkin D.W. Pfaff Preproenkephalin promoter yields region-specific and long-term expression in adult brain after direct in vivo gene transfer via a defective herpes simplex viral vector Proc. Natl. Acad. Sci. USA 91 1994 8979-8983
9. B.K. Jin et. al. Prolonged in vivo gene expression driven by a tyrosine hydroxylase promoter in a defective herpes simplex virus amplicon vector Hum. Gene Ther. 7 1996 2015-2024
10. A. Dobson T.P. Margolis F. Sedarati J. Stevens L.T. Feldman A latent, nonpathogenic HSV-1 derived vector stably expresses ́galactosidase in mouse neurons Neuron 5 1990 353-360
11. D.L. Choi-Lundberg et. al. Dopaminergic neurons protected from degeneration by GDNF gene therapy Science 275 1997 838-841
12. M.S. Szczypka R.J. Mandel D.A. Donahue R.O. Snyder S.E. Leff R.D. Palmiter Viral gene delivery selectively restores feeding and prevents lethality of dopamine-deficient mice Neuron 22 1999 167-178
13. J. Ellis D. Pannell The beta-globin locus control region versus gene therapy vectors: A struggle for expression Clin. Genet. 59 2001 17-24
14. D. Pannell J. Ellis Silencing of gene expression: Implications for design of retrovirus vectors Rev. Med. Virol. 11 2001 205-217
15. Z. Ivics P.B. Hackett R.H. Plasterk Z. Izsvak Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells Cell 91 1997 501-510
16. S.R. Yant L. Meuse W. Chiu Z. Ivics Z. Izsvak M.A. Kay Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system Nat. Genet. 25 2000 35-41
17. G. Luo Z. Ivics Z. Izsvak A. Bradley Chromosomal transposition of a Tc1/ mariner-like element in mouse embryonic stem cells Proc. Natl. Acad. Sci. USA 95 1998 10769-10773
18. B. Lu H.J. Federoff Herpes simplex virus type 1 amplicon vectors with glucocorticoid-inducible gene expression Hum. Gene Ther. 6 1995 421-430
19. G.J. Brewer Serum-free B27/neurobasal medium supports differentiated growth of neurons from the striatum, substantia nigra, septum, cerebral cortex, cerebellum, and dentate gyrus J. Neurosci. Res. 42 1995 674-683
20. A.I. Geller K. Keyomarsi J. Bryan A.B. Pardee An efficient deletion mutant packaging system for defective herpes simplex virus vectors: potential applications to human gene therapy and neuronal physiology Proc. Natl. Acad. Sci. USA 87 1990 8950-8954
21. W.J. Bowers D.F. Howard A.I. Brooks M.W. Halterman H.J. Federoff Expression of vhs and VP16 during HSV-1 helper virus-free amplicon packaging enhances titers Gene Ther. 8 2001 111-120
22. W.J. Bowers D.F. Howard H.J. Federoff Discordance between expression and genome transfer titering of HSV amplicon vectors: Recommendation for standardized enumeration Mol. Ther. 1 2000 294-299
23. T. Nomoto et. al. Distinct patterns of gene transfer to gerbil hippocampus with recombinant adeno-associated virus type 2 and 5 Neurosci. Lett. 340 2003 153-157
24. R.L. Smith D.L. Traul J. Schaack G.H. Clayton K.J. Staley C.L. Wilcox Characterization of promoter function and cell-type-specific expression from viral vectors in the nervous system J. Virol. 74 2000 11254-11261
25. F. Beermann E. Hummler E. Schmid G. Schutz Perinatal activation of a tyrosine aminotransferase fusion gene does not occur in albino lethal mice Mech. Dev. 42 1993 59-65
26. C.H. Yeh A.J. Shatkin A cis-acting element in Rous sarcoma virus long terminal repeat required for promoter repression by HeLa nuclear protein p21 J. Biol. Chem. 270 1995 15815-15820
27. C. Laker et. al. Host cis-mediated extinction of a retrovirus permissive for expression in embryonal stem cells during differentiation J. Virol. 72 1998 339-348
28. 4+ T cells J. Immunol. 159 1997 3383-3390
29. M.B. Luskin Mammalian forebrain development: How cells find a permanent home and establish their identity J. NIH Res. 5 1993 60-64
30. M.E. Hatten Central nervous system neuronal migration Annu. Rev. Neurosci. 22 1999 511-539
31. P. Grandi et. al. HSV-1 virions engineered for specific binding to cell surface receptors Mol. Ther. 9 2004 419-427
32. Z. Izsvak Z. Ivics Sleeping Beauty transposition: Biology and applications for molecular therapy Mol. Ther. 9 2004 147-156
33. H. Zayed Z. Izsvak D. Khare U. Heinemann Z. Ivics The DNA-bending protein HMGB1 is a cellular cofactor of Sleeping Beauty transposition Nucleic Acids Res. 31 2003 2313-2322