Transduction of brain by herpes simplex virus vectors

Molecular Therapy

Volumn 15, Issue 1, 2007, Pages 20-29

  Berges, Bradford K ^a   Wolfe, John H ^a   Fraser, Nigel W ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENE PRODUCT; LYSOSOME ENZYME; VIRUS PROTEIN;

ADENOVIRUS VECTOR; BLOOD BRAIN BARRIER; BRAIN DISEASE; BRAIN REGION; BRAIN TUMOR; CLINICAL PRACTICE; CYTOTOXICITY; EXPERIMENTAL MODEL; EXPERIMENTATION; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HERPES SIMPLEX VIRUS 1; LATENT VIRUS INFECTION; LENTIVIRUS VECTOR; LYSOSOME STORAGE DISEASE; MEDICAL LITERATURE; MEMBRANE PERMEABILITY; NERVE CELL; NERVE CELL LESION; NERVOUS SYSTEM; NONHUMAN; PARKINSON DISEASE; PARVOVIRUS VECTOR; REPORTER GENE; RETROVIRUS VECTOR; REVIEW; SAFETY; SCALE UP; STRAIN DIFFERENCE; TRANSPORT KINETICS; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS STRAIN; VIRUS TRANSMISSION; VIRUS VIRULENCE;

ANIMALS; BIOLOGICAL TRANSPORT; BRAIN; GENETIC VECTORS; HUMANS; NEURONS; SIMPLEXVIRUS; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; HUMAN HERPESVIRUS 1; LENTIVIRUS; SIMPLEXVIRUS;

EID: 33846015516     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300018     Document Type: Review

References (121)

1. Kremer, EJ (2005). Gene transfer to the central nervous system: Current state of the art of the viral vectors. Curr Genomics 6: 13-37.
2. Lowenstein, PR and Castro, MG (2002). Progress and challenges in viral vector-mediated gene transfer to the brain. Curr Opin Mol Ther 4: 359-371.
3. Ghodsi, A et al. (1998). Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain. Hum Gene Ther 9: 2331-2340.
4. Ohashi, T et al. (1997). Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc Natl Acad Sci USA 94: 1287-1292.
5. Stein, CS, Ghodsi, A, Derksen, T and Davidson, BL (1999). Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice. J Virol 73: 3424-3429.
6. Bosch, A, Perret, E, Desmaris, N and Heard, JM (2000). Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors. Mol Ther 1: 63-70.
7. Elliger, SS, Elliger, CA, Aguilar, CP, Raju, NR and Watson, GL (1999). Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector. Gene Ther 6: 1175-1178.
8. Elliger, SS, Elliger, CA, Lang, C and Watson, GL (2002). Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice. Mol Ther 5: 617-626.
9. Frisella, WA et al. (2001). Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Mol Ther 3: 351-358.
10. Passini, MA, Lee, EB, Heuer, GG and Wolfe, JH (2002). Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. J Neurosci 22: 6437-6446.
11. Sferra, TJ et al. (2000). Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysac-charidosis mouse. Hum Gene Ther 11: 507-519.
12. Skorupa, AF, Fisher, KJ, Wilson, JM, Parente, MK and Wolfe, JH (1999). Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis mice. Exp Neurol 160: 17-27.
13. Bosch, A, Perret, E, Desmaris, N, Trono, D and Heard, JM (2000). Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum Gene Ther 11: 1139-1150.
14. Brooks, AI et al. (2002). Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors. Proc Natl Acad Sci USA 99: 6216-6221.
15. Passini, MA et al. (2003). Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice. J Virol 77: 7034-7040.
16. Pardridge, WM (2005). The blood-brain barrier: Bottleneck in brain drug development. NeuroRx 2: 3-14.
17. Mazarakis, ND et al. (2001). Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Hum Mol Genet 10: 2109-2121.
18. Passini, MA et al. (2005). AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease. Mol Ther 11: 754-762.
19. Cearley, CN and Wolfe, JH (2006). Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther 13: 528-537.
20. Vite, CH, Passini, MA, Haskins, ME and Wolfe, JH (2003). Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther 10: 1874-1881.
21. Moore, DJ, West, AB, Dawson, VL and Dawson, TM (2005). Molecular pathophysiology of Parkinson's disease. Ann Rev Neurosci 28: 57-87.
22. Ellinwood, NM, Vite, CH and Haskins, ME (2004). Gene therapy for lysosomal storage diseases: The lessons and promise of animal models. J Gene Med 6: 481-506.
23. Chen, MY et al. (2005). Surface properties, more than size, limiting convective distribution of virus-sized particles and viruses in the central nervous system. J Neurosurg 103: 311-319.
24. Begley, DJ (2004). Delivery of therapeutic agents to the central nervous system: The problems and the possibilities. Pharmacol Ther 104: 29-45.
25. Peltekian, E, Parrish, E, Bouchard, C, Peschanski, M and Lisovoski, F (1997). Adenovirus-mediated gene transfer to the brain: Methodological assessment. J Neurosci Meth 71: 77-84.
26. Teschemacher, AG et al. (2005). Targeting specific neuronal populations using adeno- and lentiviral vectors: Applications for imaging and studies of cell function. Exp Physiol 90: 61-69.
27. McCown, TJ (2005). Adeno-associated virus (AAV) vectors in the CNS. Curr Gene Ther 5: 333-338.
28. Glorioso, JC and Fink, DJ (2004). Herpes vector-mediated gene transfer in treatment of diseases of the nervous system. Annu Rev Microbiol 58: 253-271.
29. Russell, WC (2000). Update on adenovirus and its vectors. J Gen Virol 81: 2573-2604.
30. Barquinero, J, Eixarch, H and Perez-Melgosa, M (2004). Retroviral vectors: New applications for an old tool. Gene Ther 11: S3-S9.
31. Kafri, T (2004). Gene delivery by lentivirus vectors - an overview. Meth in Mol Biol 246: 367-390.
32. McCarty, DM, Young, SM and Samulski, RJ (2004). Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Ann Rev Genet 38: 819-845.
33. Cook, ML and Stevens, JG (1973). Pathogenesis of herpetic neuritis and ganglionitis in mice: Evidence for intra-axonal transport of infection. Infect Immun 7: 272-288.
34. Passini, MA and Wolfe, JH (2001). Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector. J Virol 75: 12382-12392.
35. Ghadge, GD et al. (1995). CNS gene delivery by retrograde transport of recombinant replication-defective adenoviruses. Gene Ther 2: 132-137.
36. Kuo, H, Ingram, DK, Crystal, RG and Mastrangeli, A (1995). Retrograde transfer of replication deficient recombinant adenovirus vector in the central nervous system for tracing studies. Brain Res 705: 31-38.
37. Wong, L-F et al. (2004). Transduction patterns of pseudotyped lentiviral vectors in the nervous system. Mol Ther 9: 101-111.
38. Kaspar, BK et al. (2002). Targeted retrograde gene delivery for neuronal protection. Mol Ther 5: 50-56.
39. Boulis, NM, Willmarth, NE, Song, DK, Feldman, EL and Imperiale, MJ (2003). Intraneural colchicine inhibition of adenoviral and adeno-associated viral vector remote spinal cord gene delivery. Neurosurgery 5: 381-387.
40. Martinov, VN et al. (2002). Targeting functional subtypes of spinal motoneurons and skeletal muscle fibers in vivo by intramuscular injection of adenoviral and adeno-associated viral vectors. Anat Embryol 205: 215-221.
41. Akli, S et al. (1993). Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 3: 224-228.
42. Kaspar, BK, Llado, J, Sherkat, N, Rothstein, JD and Gage, FH (2003). Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301: 839-842.
43. Kristensson, K, Lycke, E and Sjostrand, J (1971). Spread of herpes simplex virus in peripheral nerves. Acta Neuropathol 17: 44-53.
44. Akhova, O, Bainbridge, M and Misra, V (2005). The neuronal host cell factor-binding protein Zhangfei inhibits herpes simplex virus replication. J Virol 79: 14708-14718.
45. Topp, KS, Meade, LB and LaVail, JH (1994). Microtubule polarity in the peripheral processes of trigeminal ganglion cells: Relevance for the retrograde transport of herpes simplex virus. J Neurosci 14: 318-325.
46. Sodeik, B, Ebersold, MW and Helenius, A (1997). Microtubule-mediated transport of incoming herpes simplex virus 1 capsids to the nucleus. J Cell Biol 136: 1007-1021.
47. Ye, GJ, Vaughan, KT, Vallee, RB and Roizman, B (2000). The herpes simplex virus 1 U(L)34 protein interacts with a cytoplasmic dynein intermediate chain and targets nuclear membrane. J Virol 74: 1355-1363.
48. Tomishima, MJ, Smith, GA and Enquist, LW (2001). Sorting and transport of alpha herpesviruses in axons. Traffic 2: 429-436.
49. Enquist, LW, Husak, PJ, Bansfield, BW and Smith, GA (1999). Infection and spread of alphaherpesviruses in the nervous system. Adv Virus Res 51: 237-347.
50. Epstein, AL, Marconi, P, Argnani, R and Manservigi, R (2005). HSV-1-derived recombinant and amplicon vectors for gene transfer and gene therapy. Curr Gene Ther 5: 445-457.
51. Lilley, CE et al. (2001). Multiple immediate-early gene-deficient herpes simplex virus vectors allowing efficient gene delivery to neurons in cell culture and widespread gene delivery to the central nervous system in vivo. J Virol 75: 4343-4356.
52. Campbell, ME, Palfreyman, JW and Preston, CM (1984). Identification of herpes simplex virus DNA sequences which encode a trans-acting polypeptide responsible for stimulation of immediate early transcription. J Mol Biol 180: 1-19.
53. Chou, J, Kern, ER, Whitley, RJ and Roizman, B (1990). Mapping of herpes simplex virus-1 neurovirulence to gamma34.5, a gene nonessential for growth in culture. Science 250: 1262-1266.
54. Kit, S and Dubbs, DR (1965). Properties of deoxythymidine kinase partially purified from noninfected and virus-infected mouse fibroblast cells. Virology 26: 16-27.
55. Forrester, A et al. (1992). Construction and properties of a mutant of herpes simplex virus type 1 with glycoprotein H coding sequences deleted. J Virol 66: 341-348.
56. Ruyechan, WT, Morse, LS, Knipe, DM and Roizman, B (1979). Molecular genetics of herpes simplex virus. II. Mapping of the major viral glycoproteins and of the genetic loci specifying the social behavior of infected cells. J Virol 29: 677-697.
57. Burton, EA, Fink, DJ and Glorioso, JC (2005). Replication-defective genomic HSV gene therapy vectors: Design, production and CNS applications. Curr Opin Mol Ther 7: 326-336.
58. Lilley, CE, Branston, RH and Coffin, RS (2001). Herpes simplex virus vectors for the nervous system. Curr Gene Ther 1: 339-358.
59. Berges, BK, Wolfe, JH and Fraser, NW (2005). Stable levels of long-term transgene expression driven by the latency-associated transcript promoter in a herpes simplex virus type 1 vector. Mol Ther 12: 1111-1119.
60. Scarpini, CG et al. (2001). Latency associated promoter transgene expression in the central nervous system after stereotaxic delivery of replication-defective HSV-1-based vectors. Gene Ther 8: 1057-1071.
61. Fraser, N, Lawrence, W, Wroblewska, Z, Gilden, D and Koprowski, H (1981). Herpes simplex virus type 1 DNA in human brain tissue. Proc Natl Acad Sci USA 78: 6461-6465.
62. Theil, D et al. (2004). Prevalence and distribution of HSV-1, VZV, and HHV-6 in human central nerve nuclei III, IV, VI, VII, and XII. J Med Virol 74: 102-106.
63. Rampling, R et al. (2000). Toxicity evaluation of replication-competent herpes simplex virus (ICP 34.5 null mutant 1716) in patients with recurrent malignant glioma. Gene Ther 7: 859-866.
64. McMenamin, MM et al. (1998). A gamma34.5 mutant of herpes simplex virus 1 causes severe inflammation in the brain. Neuroscience 83: 1225-1237.
65. Kesari, S, Lee, VM-Y, Brown, SM, Trojanoski, JQ and Fraser, NW (1996). Selective vulnerability of mouse CNS neurons to latent infection with a neuroattenuated herpes simplex virus-1. J Neurosci 16: 5644-5653.
66. Spivack, JG and Fraser, NW (1987). Detection of herpes simplex virus type 1 transcripts during latent infection in mice. J Virol 61: 3841-3847.
67. Lachmann, RH and Efstathiou, S (1997). Utilization of the herpes simplex virus type 1 latency-associated regulatory region to drive stable reporter gene expression in the nervous system. J Virol 71: 3197-3207.
68. Maidment, NT et al. (1996). Expression of the lacZ reporter gene in the rat basal forebrain, hippocampus, and nigrostriatal pathway using a nonreplicating herpes simplex vector. Exp Neurol 139: 107-114.
69. McMenamin, MM et al. (1998). Potential and limitations of a gamma-34.5 mutant of herpes simplex 1 as a gene therapy vector in the CNS. Gene Ther 5: 594-604.
70. Rogers, JH et al. (2003). A herpesvirus vector can transduce axotomized brain neurons. Exp Neurol 183: 548-558.
71. Shiraki, K et al. (1998). A live non-neurovirulent herpes simplex virus vector expresses beta-galactosidase in the nervous system of the Wistar and Sprague-Dawley strain rat for a prolonged period. Neurosci Lett 245: 69-72.
72. Smith, C, Lachmann, RH and Efstathiou, S (2000). Expression from the herpes simplex virus type 1 latency-associated promoter in the murine central nervous system. J Gen Virol 81: 649-662.
73. Wolfe, JH, Deshmane, SL and Fraser, NW (1992). Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice. Nat Genet 1: 379-384.
74. Wood, MJA et al. (1994). Specific patterns of defective HSV-1 gene transfer in the adult central nervous system: Implications for gene targeting. Exp Neurol 130: 127-140.
75. Zhu, J, Kang, W, Wolfe, JH and Fraser, NW (2000). Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector. Mol Ther 2: 82-94.
76. Wood, MJ, Byrnes, AP, Pfaff, DW, Rabkin, SD and Charlton, HM (1994). Inflammatory effects of gene transfer into the CNS with defective HSV-1 vectors. Gene Ther 1: 283-291.
77. Yang, T et al. (2001). Enhanced reporter gene expression in the rat brain from helper virus-free HSV-1 vectors packaged in the presence of specific mutated HSV-1 proteins that affect the virion. Mol Brain Res 90: 1-16.
78. Tabbaa, S et al. (2005). In vivo gene transfer to the brain cortex using a single injection of HSV-1 vector into the medial septum. Folia Morphologica (Warszawa) 64: 273-281.
79. Broberg, EK et al. (2004). Spread and replication of and immune response to gamma 34.5-negative herpes simplex virus type 1 vectors in BALB/c mice. J Virol 78: 13139-13152.
80. Huang, Q et al. (1992). Introduction of a foreign gene (Escherichia coli lacZ) into rat neostriatal neurons using herpes simple virus mutants: A light and electron microscopic study. Exp Neurol 115: 303-316.
81. Coffin, RS, MacLean, AR, Latchman, DS and Brown, SM (1996). Gene delivery to the central and peripheral nervous systems of mice using HSV1 ICP34.5 deletion mutant vectors. Gene Ther 3: 886-891.
82. Margolis, TP, Sedarati, F, Dobson, AT, Feldman, LT and Stevens, JG (1992). Pathways of viral gene expression during acute neuronal infection with HSV-1. Virology 189: 150-160.
83. Margolis, TP, Bloom, DC, Dobson, AT, Feldman, LT and Stevens, JG (1993). Decreased reporter gene expression during latent infection with HSV LAT promoter constructs. Virology 197: 585-592.
84. Marshall, KR, Lachmann, RH, Efstathiou, S, Rinaldi, A and Preston, CM (2000). Long-term transgene expression in mice infected with a herpes simplex virus type 1 mutant severely impaired for immediate-early gene expression. J Virol 74: 956-964.
85. Palmer, JA et al. (2000). Development and optimization of herpes simplex virus vectors for multiple long-term gene delivery to the peripheral nervous system. J Virol 74: 5604-5618.
86. Zemanick, MC, Strick, PL and Dix, RD (1991). Direction of transneuronal transport of herpes simplex virus 1 in the primate motor system is strain-dependent. Proc Natl Acad Sci USA 88: 8048-8051.
87. Bak, IJ, Markham, CH, Cook, ML and Stevens, JG (1978). Ultrastructural and immunoperoxidase study of striatonigral neurons by means of retrograde axonal transport of herpes simplex virus. Brain Res 143: 361-368.
88. Barnett, EM, Cassell, M and Perlman, S (1993). Two neurotropic viruses, herpes simplex virus type 1 and mouse hepatitis virus, spread along different neural pathways from the main olfactory bulb. J Neurosci 57: 1007-1025.
89. Berges, BK et al. (2006). Widespread correction of lysosomal storage in the mucopolysaccharidosis type VII mouse brain with a herpes simplex type virus 1 vector expressing beta-glucuronidase. Mol Ther 13: 859-869.
90. Rodahl, E and Haarr, L (2000). A herpes simplex virus type 1 vector as marker for retrograde neuronal tracing: Characterization of lacZ transcription and localization of labelled neuronal cells in sensory and autonomic ganglia after inoculation of the anterior segment of the eye. Exp Eye Res 71: 495-501.
91. Huang, QS, Valyi-Nagy, T, Kesari, S and Fraser, NW (1997). Beta-Gal enzyme activity driven by the HSV LAT promoter does not correspond to beta-gal RNA levels in mouse trigeminal ganglia. Gene Ther 4: 797-807.
92. Laquerre, S et al. (1998). Heparan sulfate proteoglycan binding by herpes simplex virus type 1 glycoproteins B and C, which differ in their contributions to virus attachment, penetration, and cell-to-cell spread. J Virol 72: 6119-6130.
93. Anderson, DB et al. (2000). Pseudotyping of glycoprotein D-deficient herpes simplex virus type 1 with vesicular stomatitis virus glycoprotein G enables mutant virus attachment and entry. J Virol 74: 2481-2487.
94. Wang, X, Kong, L, Zhang, GR, Sun, M and Geller, AI (2005). Targeted gene transfer to nigrostriatal neurons in the rat brain by helper virus-free HSV-1 vector particles that contain either a chimeric HSV-1 glycoprotein C-GDNF or a gC-BDNF protein. Brain Res Mol Brain Res 139: 88-102.
95. Perez, MC, Hunt, SP, Coffin, RS and Palmer, JA (2004). Comparative analysis of genomic HSV vectors for gene delivery to motor neurons following peripheral inoculation in vivo. Gene Ther 11: 1023-1032.
96. Labetoulle, M et al. (2003). HSV-1 latency sites after inoculation in the lip: Assessment of their localization and connections to the eye. Invest Ophthalmol Vis Sci 44: 217-225.
97. Maggioncalda, J, Mehta, A, Su, YH, Fraser, NW and Block, TM (1996). Correlation between herpes simplex virus type 1 rate of reactivation from latent infection and the number of infected neurons in trigeminal ganglia. Virology 225: 72-81.
98. Wang, K, Lau, TY, Morales, M, Mont, EK and Straus, SE (2005). Laser-capture microdissection: Refining estimates of the quantity and distribution of latent herpes simplex virus 1 and varicella-zoster virus DNA in human trigeminal Ganglia at the single-cell level. J Virol 79: 14079-14087.
99. Sun, M et al. (2004). Coexpression of tyrosine hydroxylase, gtp cyclohydrolase I, aromatic amino acid decarboxylase, and vesicular monoamine transporter 2 from a helper virus-free herpes simplex virus type 1 vector supports high-level, long-term biochemical and behavioral correction of a rat model of parkinson's disease. Hum Gene Ther 15: 1177-1196.
100. Kesari, S et al. (1995). Therapy of experimental human brain tumors using a neuroattenuated herpes simplex virus mutant. Lab Invest 73: 636-648.
101. Bloom, DC et al. (1995). Long-term expression of a reporter gene from latent herpes simplex virus in the rat hippocampus. Mol Brain Res 31: 48-60.
102. Kordower, JH et al. (1999). Lentiviral gene transfer to the nonhuman primate brain. Exp Neurol 160: 1-16.
103. Lawrence, MS et al. (1999). Inflammatory responses and their impact on beta-galactosidase transgene expression following adenovirus vector delivery to the primate caudate nucleus. Gene Ther 6: 1368-1379.
104. Vite, CH et al. (2005). Effective gene therapy for an inherited CNS disease in a large animal model. Ann Neurol 57: 355-364.
105. McLean, JH, Shipley, MT and Bernstein, DI (1989). Golgi-like, transneuronal retrograde labeling with CNS injections of herpes simplex virus type 1. Brain Res Bull 22: 867-881.
106. Lynch, JC, Hoover, JE and Strick, PL (1994). Input to the primate frontal eye field from the substantia nigra, superior colliculus, and dentate nucleus demonstrated by transneuronal transport. Exp Brain Res 100: 181-186.
107. Springer, SL et al. (2001). Infection and establishment of latency in the dog brain after direct inoculation of a nonpathogenic strain of herpes simplex virus-1. J Neurovirol 7: 149-154.
108. Meikle, PJ, Hopwood, JJ and Clague, AE (1999). Prevalence of lysosomal storage disorders. J Am Med Assoc 281: 249-254.
109. Vellodi, A (2005). Lysosomal storage disorders. Br J Haematol 128: 413-431.
110. Frantantoni, JC, Hall, CW and Neufeld, EF (1968). Hurler and Hunter syndromes: Mutual correction of the defect in cultured fibroblasts. Science 162: 570-572.
111. O'Brien, JS, Miller, AL, Loverde, AW and Veath, ML (1973). Sanfilippo disease type B: Enzyme replacement and metabolic correction in cultured fibroblasts. Science 181: 753-755.
112. Brot, FE, Glaser, JH, Roozen, KJ, Sly, WS and Stahl, PD (1974). In vitro correction of deficient human fibroblasts by beta-glucuronidase from different human sources. Biochem Biophys Res Commun 57: 1-8.
113. Hennig, AK et al. (2003). Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice. J Neurosci 23: 3302-3307.
114. Luca, T et al. (2005). Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery. Mol Ther 12: 669-679.
115. Griffey, M, Macauley, SL, Ogilvie, JM and Sands, MS (2005). AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis. Mol Ther 12: 413-421.
116. Wolfe, JH et al. (1992). Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 360: 749-753.
117. Moullier, P, Bohl, D, Heard, JM and Danos, O (1993). Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts. Nat Genet 4: 154-159.
118. Puskovic, V et al. (2004). Prolonged biologically active transgene expression driven by HSV LAP2 in brain in vivo 10: 1.
119. Markert, JM et al. (2000). Conditionally replicating herpes simplex virus mutant, G207 for the treatment of malignant glioma: Results of a phase I trial. Gene Ther 7: 867-874.
120. Pardridge, WM (2003). Blood-brain barrier drug targeting: The future of brain drug development. Mol Interv 3: 90-105.
121. Neve, RL, Neve, KA, Nestler, EJ and Carlezon, WAJ (2005). Use of herpes virus amplicon vectors to study brain disorders. Biotechniques 39: 381-391.