25. Treatment of GM2 gangliosidosis: Past experiences, implications, and future prospects

Advances in Genetics

Volumn 44, Issue , 2001, Pages 317-339

  Rattazzi, Mario C ^a   Dobrenis, Kostantin ^b  

^a NEW YORK STATE INSTITUTE FOR BASIC RESEARCH IN DEVELOPMENTAL DISABILITIES   (United States)

^b ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE;

ANIMAL; BONE MARROW TRANSPLANTATION; BRAIN; CAT; DISEASE MODEL; GENE THERAPY; GENETICS; GM2 GANGLIOSIDOSIS; HUMAN; METABOLISM; METHODOLOGY; NERVE CELL; REVIEW;

ANIMALS; BETA-N-ACETYLHEXOSAMINIDASE; BONE MARROW TRANSPLANTATION; BRAIN; CATS; DISEASE MODELS, ANIMAL; GANGLIOSIDOSES, GM2; GENE THERAPY; HUMANS; NEURONS;

EID: 0035228743     PISSN: 00652660     EISSN: None     Source Type: Book Series    
DOI: 10.1016/s0065-2660(01)44089-2     Document Type: Review

References (115)

1. B. Andersen-Beckh T. Binz H. Kurazono T. Mayer U. Eisel P. Niemann Expression of tetanus toxin subfragments in vitro and characterization of epitopes Infect. Immun. 57 1989 3498 3505
2. J.M. Anderson C.M. Van Itallie Tight junctions and the molecular basis for regulation of paracellular permeability Am. J. Physiol. 269 1995 G467 G475
3. G. Ashwell A. Morell Role of surface carbohydrates in the hepatic recognition and transport of circulating glycoproteins Adv. Enzymol. 41 1974 99 128
4. B. Bizzini K. Stoeckel M. Schwab An antigenic polypeptide fragment isolated from Tetanus toxin: Chemical characterization, binding to ganglioside and retrograde axonal transport in various nervous systems J. Neurochem. 28 1977 529 542
5. B. Bizzini R. Akert M. Glicksman P. Grob Preparation of conjugates using two tetanus toxin derived fragments: Their binding to gangliosides and isolated synaptic membranes and their immunological properties Toxicon 18 1980 561 572
6. R.H. Bobo D.W. Laske A. Akbasak P.F. Morrison R.L. Dedrick E.H. Oldfield Convection-enhanced delivery of macromolecules into the brain Proc. Natl. Acad. Sci. (USA) 91 1994 2076 2080
7. T. Boulikas Nuclear localization signals (NLS) Crit. Rev. Eukaryot. Gene Expr. 3 1993 193 227
8. K.E. Bove C. Daugherty G.A. Grabowski Pathological findings in Gaucher disease type 2 patients following enzyme therapy Hum. Pathol. 26 1995 193 207
9. M.W. Brightman The distribution within the brain of ferritin injected into cerebral fluid compartments Am. J. Anat. 117 1965 193 220
10. M.W. Brightman T.S. Reese Junctions between intimately apposed cell membranes in the vertebrate brain J. Cell Biol. 40 1969 618 677
11. R.D. Broadwell M.W. Brightman Entry of peroxidase into neurons of the central and peripheral nervous system from extracerebral and cerebral blood J. Comp. Neurol. 166 1976 257 284
12. J.B. Cabot A. Menneone N. Bogan J. Carroll C. Evinger J.T. Erichsen Retrograde, transsynaptic and transneuronal transport offragment C of tetanus toxin by sympathetic preganglionic neurons Neuroscience 40 1991 805 823
13. M2 ganglioside storage disease in cats with β-hexosaminidase deficiency Science 196 1977 1014 1017
14. M.E. Couce A.J. Weatherington J.F. McGinty Expression of insulin-like growth factor-II (IGF-II) and IGF-II/mannose-6-phosphate receptor in the rat hippocampus: An in situ hybridization and immunocytochemical study Endocrinology 131 1992 1636 1642
15. H.F. Cserr Role of secretion and bulk flow of brain interstitial fluid in brain volume regulation Ann. N.Y. Acad. Sci. 529 1988 9 20
16. H.F. Cserr P.M. Knopf Cervical lymphatics, the blood-brain barrier and the immunore-activity of the brain: A new view Immunol. Today 13 1992 507 512
17. B.L. Davidson C.S. Stein J.A. Heth I. Martins R.M. Kotin T.A. Derksen J. Zabner A. Ghodsi J.A. Chiorini Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system Proc. Natl. Acad. Sci. U.S.A. 97 2000 3428 3432
18. C. de Duve From cytases to lysosomes Fed. Proc. 23 1964 1045 1049
19. E. Dejana M. Corada M.G. Lampugnani Endothelial cell-to-cell junctions FASEB J. 1995 910 918
20. N. Desnick P.D. Snyder S.J. Desnick W. Krivit H.L. Sharp Sandhoff's disease: Ultrastructural and biochemical studies B.W. Volk S.M. Aronson Sphingolipid, Sphingolipidosis and Allied Disorders 1972 Plenum New York 351 371
21. K. Dobrenis M.C. Rattazzi Enhanced uptake of human β-hexosaminidase A (Hex A) by CNS neurons in vitro Soc. Neurosci. Abst. 13 3 1987 1685
22. K. Dobrenis M.C. Rattazzi Enzyme replacement in cultured neurons with tetanus toxin fragment C (TTC)-β-hexosaminidase A (Hex A) conjugate Pediatr. Res. 23 1988 551A
23. K. Dobrenis P. Schwartz A. Joseph M.C. Rattazzi Neuronal enzyme replacement in vitro using tetanus toxin fragment C (TTC)-conjugated-hexosaminidase A (Hex A) Am. J. Hum. Genet. 41 1987 A5
24. K. Dobrenis A. Joseph M.C. Rattazzi Neuronal lysosomal enzyme replacement using fragment C of tetanus toxin Proc. Natl. Acad. Sci. (USA) 89 1992 2297 2301
25. K. Dobrenis D.A. Wenger S.U. Walkley Extracellular release of lysosomal glycosidases in cultures of cat microglia Mol. Biol. Cell. 5 1994 113A (Suppl.)
26. 3 receptor in mammalian microglia, astrocytes and Kupffer cells Brain Res. 686 1995 239 248
27. K. Dobrenis P.S. Finamore R. Masui S.U. Walkley Secretion of lysosomal glycosidases by microglia in culture Mol. Biol. Cell 7 1996 325A (Suppl.)
28. N.D. Doolittle M.E. Miner W.A. Hall T. Siegal E. Jerome E. Osztie L.D. McAllister J.S. Bubalo D.F. Kraemer D. Fortin R. Nixon L.L. Muldoon E.A. Neuwelt Safety and efficacy of a multicenter study using intraarterial chemotherapy in conjunction with osmotic opening of the blood-brain barrier for the treatment of patients with malignant brain tumors Cancer 88 2000 637 647
29. S.E. Doran X.D. Ren A.L. Betz A.L. Pagel E.A. Neuwelt B.J. Roessler B.L. Davidson Gene expression from recombinant viral vectors in the central nervous system after blood-brain barrier disruption Neurosurgery 36 1995 965 970
30. D.D. Dunlap A. Maggi M.R. Soria L. Monaco Nanoscopic structure of DNA condensed for gene delivery Nucl. Acid Res. 25 1997 3095 3101
31. P. Erbacher A.C. Roche M. Monsigny P. Midoux Putative role of chloroquine in gene transfer into a human hepatoma cell line by DNA/lactosylated polylysine complexes Exp. Cell. Res. 225 1996 186 194
32. Z. Fabry C.S. Raine M.C. Hart Nervous tissue as an immune compartment: The dialect of the immune response in the CNS Immunol. Today 15 1994 218 224
33. P.S. Fishman D.R. Carrigan Retrograde transneuronal transfer of the C-fragment of tetanus toxin Brain Res. 406 1987 275 279
34. J.W. Francis B.A. Hosler R.H. Brown Jr P.S. Fishman CuZn superoxide dismutase (SOD-1): Tetanus toxin fragment C hybrid protein for targeted delivery of SOD-1 to neuronal cells J. Biol. Chem. 270 1995 15434 154342
35. J.C. Fratantoni C.W. Hall E.F. Neufeld Hurler and Hunter syndromes. Mutual correction of the defect in cultured fibroblasts Science 162 1968 570 571
36. B. Geiger R. Arnon Hexosaminidases A and B from human placenta Meth. Enzymol. 50(C) 1978 547 555
37. B. Geiger B.U. von Specht R. Arnon Stabilization of human-D-N-Acetyl hexosaminidase A towards proteolytic inactivation by coupling it to poly (N-vinyl pyrrolidone Eur. J. Biochem. 73 1977 141 147
38. J. Glaser K.J. Rosen P.E. Brut W.S. Sly Multiple isoelectric and recognition forms of human beta glucuronidase activity Arch. Biochem. Biophys. 166 1975 536 542
39. V. Godel M. Bluhmenthal B. Goldman G. Keven B. Padeh Visual functions in Tay-Sachs diseased patients following enzyme replacement therapy Metab. Ophthalmol. 2 1978 27 32
40. L.B. Gordon P.M. Knopf H.F. Cserr Ovalbumin is more immunogenic when introduced into brain or cerebrospinal fluid than into extracerebral sites J. Neuroimmunol. 40 1992 81 87
41. G.A. Grabowski N.W. Barton G. Pastores J.M. Dambrosia T.K. Banerjee M.A. McKee C. Parker R. Schiffmann S.C. Hill R.O. Brady Enzyme therapy in type 1 Gaucher disease: Comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources Ann. Intern. Med. 122 1995 33 39
42. J.H. Grubb J.W. Kyle L.B. Cody W.S. Sly Large scale purification of phosphorylated recombinant human b-glucuronidase from overexpressing mouse L cells FASEB J. 7 1993 A1255
43. M.K. Gumerlock B.D. Belshe R. Madsen C. Watts Osmotic blood-brain barrier disruption and chemotherapy in the treatment of high grade malignant glioma: Patient series and literature review J. Neurooncol. 12 1992 33 46
44. W.F. Hickey Migration of hematogenous cells through the blood-brain barrier and the initiation of CNS inflammation Brain Pathol. 1 1991 97 105
45. D.F. Hill P.N. Bullock F. Chiappelli L.H. Rome Binding and internalization of lysosomal enzymes by primary cultures of rat glia J. Neurosci. Res. 14 1985 35 47
46. T. Hirase J.M. Staddon M. Saitou M. Ando-Akatsuka Itoh M. Furuse K. Fujimoto S. Tsukita L.L. Rubin Occludin as a possible determinant of tight junction permeability in endothelial cells J. Cell Sci. 110 1997 1603 1610
47. P.M. Hoogerbrugge O.F. Brouwer P. Bordigoni O. Ringden P. Kapaun J.J. Ortega A. O'Meara G. Cornu G. Souillet D. Frappaz Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation Lancet 345 8962 1995 1398 1402
48. Y.A. Ioannou Gene therapy for lysosomal storage disorders with neuropathology J. Am. Soc. Nephro. 11 2000 1542 1547
49. Y.A. Ioannou D.F. Bishop R.J. Desnick Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion J. Cell. Biol. 119 1992 1137 1150
50. W. Jessup R.T. Dean Secretion by mononuclear phagocytes of lysosomal hydrolases bearing ligands for the mannose-6-phosphate receptor system of fibroblasts: Evidence for a second mechanism of spontaneous secretion? Biochem. Biophys. Res. Commun. 105 1982 922 927
51. M. Jeyakumar F. Norflus C.J. Tifft M. Cortina-Borja T.D. Butters R.L. Proia V.H. Perry R.A. Dwek F.M. Platt Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation Blood 97 2001 327 329
52. W.A. Johnson R.J. Desnick D.M. Lang H.L. Sharp W. Krivit R. Brady R.O. Brady Intravenous injection of purified hexosaminidase A into a patient with Tay-Sachs disease D. Bergsma Enzyme Therapy in Genetic Diseases 1973 Williams & Wilkins Baltimore 120 124
53. E.D. Kakkis A. Matynia A.J. Jonas E.F. Neufeld Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells Protein Exp. Purif. 5 1993 225 232
54. M.G. Kaplitt P. Leone R.J. Samulski X. Xiao D.W. Pfaff K.L. O'Malley M.J. During Long-term gene expression and phenotypic correction using adeno-associated virus vectors in mammalian brain Nature Genetics 8 1994 148 153
55. M.G. Kaplitt H. Makimura Defective viral vectors as agents for gene transfer in the nervous system J. Neurosci. Methods 71 1997 125 132
56. S. Kar J.G. Chabot R. Quirion Quantitative autoradiographic localization of [125I] insulin-like growth factor I, [125I] insulin-like growth factor II, and [125I] insulin receptor binding sites in developing and adult rat brain J. Comp. Neurol. 333 1993 375 397
57. A. Knight J. Carvajal H. Schneider C. Coutelle S. Chamberlain N. Fairweather Non-viral neuronal gene delivery mediated by the HC fragment of tetanus toxin Eur. J. Biochem. 259 1999 762 769
58. J.W. Kusiak J.M. Toney J.M. Quirk R.O. Brady Specific binding of 125 I-labeled b-hexosaminidase A to rat brain synaptosomes Proc. Natl. Acad. Sci. (USA) 76 1979 982 985
59. D.W. Laske R.J. Youle E.H. Oldfield Tumor regression with regional distribution of the targeted toxin TF-CRM107 in patients with malignant brain tumors Nature Medicine 3 1997 1362 1368
60. W. Li H.G.P. Ryser W.C. Shen Altered endocytosis in a mutant of LM fibroblasts defective in cell-cell fusion J. Cell Physiol. 126 1986 161 166
61. D.M. Lieberman D.W. Laske P.F. Morrison K.S. Bankiewicz E.H. Oldfield Convection-enhanced distribution of large molecules in gray matter during interstitial drug infusion J. Neurosurg. 82 1995 1021 1102
62. L.D. McAllister N.D. Doolittle P.E. Guastadisegni D.F. Kraemer C.A. Lacy J.R. Crossen E.A. Neuwelt Cognitive outcome and long-term follow-up results after enhanced chemotherapy delivery for primary central nervous system lymphoma Neurosurgery 46 2000 51 60
63. T.J. McCown X. Xiao J. Li G.R. Breese R.J. Samulski Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector Brain Res. 713 1996 99 107
64. K. Møllgard N.R. Saunders The development of the human blood-brain and blood-CSF barrier Neuropathol. Appl. Neurobiol. 12 1986 337 358
65. T. Moos K. Møllgard Cerebrovascular permeability to azo dyes and plasma proteins in rodents of different ages Neuropathol. Appl. Neurobiol. 19 1993 120 127
66. E.J. Natoli P.O. Livingston C.S. Pukel K.O. Lloyd H. Wiegandt J. Szalsy H.F. Oettgen L.J. Old A murine monoclonal antibody detecting N-acetyl-and N-glycolyl-GM2: Characterization of cell surface reactivity Cancer Res. 46 1986 4116 4120
67. F.C. Nielsen S. Gammeltoft Mannose-6-phosphate stimulates proliferation of neuronal precursor cells FEBS Lett. 262 1990 142 144
68. P. Nissley W. Kiess M. Sklar Developmental expression of the IGF-II/mannose 6-phosphate receptor Mol. Reprod. Dev. 35 1993 408 413
69. F. Norflus C. Tifft M.P. McDonald G. Goldstein J.N. Crawley A. Hoffmann K. Sandhoff K. Suzuki R.L. Proia Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice J. Clin. Invest. 101 1998 1881 1888
70. K. Ohata A. Marmarou J.T. Povlishock An immunocytochemical study of protein clearance in brain infusion edema Acta. Neuropathol. (Berl.) 81 1990 162 177
71. D.C. O'Neil W.R. Bartholomew M.C. Rattazzi Antigenic homology of feline and human-hexosaminidase Biochim. Biophys. Acta 580 1979 1 9
72. Y. Oya R.L. Proia F. Norflus C.J. Tifft C. Langaman K. Suzuki Distribution of enzyme-bearing cells in GM2 gangliosidosis mice: regionally specific pattern of cellular infiltration following bone marrow transplantation Acta Neuropathol. (Berl) 99 2000 161 168
73. J.C. Perales G.A. Grossmann M. Molas G. Liu T. Ferkol J. Harpst H. Oda R.W. Hanson Biochemical and functional characterization of DNA complexes capable of targeting genes to hepatocytes via the asialoglycoprotein receptor J. Biol. Chem. 272 1997 7398 7407
74. V.H. Perry Macrophages and the Nervous System 1994 Landes Austin, TX 6 26
75. S.E. Pontow V. Kery P.D. Stahl Mannose receptor Int. Rev. Cytol. 137B 1992 221 244
76. S.I. Rapoport Blood-Brain Barrier in Physiology and Medicine 1976 Raven New York
77. S.I. Rapoport Osmotic opening of the blood-brain barrier: principles, mechanisms and therapeutic applications Cell Mol. Neurobiol. 20 2000 217 230
78. M.C. Rattazzi β-Hexosaminidase isozymes and replacement therapy in GM2 gangliosidosis Isozymes: Curr. Top. Biol. Med. Res. 11 1983 65 81
79. M. Rattazzi K. Dobrenis Enzyme replacement: Overview and prospects R.J. Desnick Therapy of Genetic Diseases 1991 Churchill-Livingstone New York 131 152
80. M.C. Rattazzi Y.A. Ioannou Fragile X syndrome: Molecular genetic approaches to therapy R. Hagerman A. Cronister Fragile X Syndrome: Diagnosis, Treatment and Research 2nd ed. 1996 Johns Hopkins Univ. Press Baltimore 412 452
81. M.C. Rattazzi H.J. Baker L.C. Cork N.R. Cox S.B. Lanse R.A. McCullough J.F. Munnell The domestic cat as a model for human GM2 gangliosidosis. Pathogenetic and therapeutic aspects F. Hommes Models for the Study of Inborn Errors of Metabolism 1979 Elsevier Amsterdam 57 72
82. M.C. Rattazzi R.A. McCullough C.J. Downing M.P. Kung Toward enzyme therapy in GM2 gangliosidosis:-Hexosaminidase infusion in normal cats Pediatr. Res. 13 1979 916 923
83. M.C. Rattazzi S.B. Lanse R.A. McCullough J.A. Nester E.A. Jacobs Towards enzyme therapy in GM2 gangliosidosis: Organ disposition and induced central nervous system uptake of human-hexosaminidase in the cat R.J. Desnick Enzyme Therapy in Genetic Diseases: 2 1980 A. R. Liss New York 179 193
84. M.C. Rattazzi A.M. Appel H.J. Baker J. Nester Toward Enzyme replacement in GM2 gangliosidosis: Inhibition of hepatic uptake of human-hexosaminidase in the cat J.W. Callahan J.A. Lowden Lysosomes and Lysosomal Storage Diseases 1981 Raven New York 405 424
85. M.C. Rattazzi A.M. Appel H.J. Baker Enzyme replacement in feline GM2 gangliosidosis: Catabolic effects of human -hexosaminidase A R.J. Desnick D.E. Patterson D.F. Scarpelli Animal Models of Inherited Metabolic Diseases 1982 A. R. Liss New York 213 220
86. M.C. Rattazzi K. Dobrenis A. Joseph P. Schwartz Modified β-D-N-Acetyl-hexosaminidase isozymes for enzyme replacement in GM2 gangliosidosis Isozymes: Curr. Top. Biol. Med.Res. 16 1987 49 65
87. M.C. Rattazzi T. Tsuda C.J. Benjamin R.E. Gordon V.L. Friedrich R.J. Desnick Y.A. Ioannou Delivery of DNA across the blood-brain barrier (BBB) Am. J. Hum. Genet. 61 4 1997 A358
88. S. Roman-Goldstein P. Mitchell J.R. Crossen P.C. Williams A. Tindall E.A. Neuwelt MR and cognitive testing of patients undergoing osmotic blood-brain barrier disruption with intraarterial chemotherapy Am. J. Neuroradiol. 16 1995 543 553
89. G.A. Rosenberg W.T. Kyner E. Estrada Bulk flow of brain interstitial fluid under normal and hyperosmolar conditions Am. J. Physiol. 283 1980 F42 F49
90. L.L. Rubin J.M. Staddon The cell biology of the blood-brain barrier Ann. Rev. Neurosci. 22 1999 11 28
91. M. Sands J.E. Barker Percutaneous intravenous injection in neonatal mice Lab. Animal Sci. 49 1999 328 330
92. M.S. Sands C. Vogler J.W. Kyle J.H. Grubb B. Levy N. Galvin W.S. Sly E.H. Birkenmeier Enzyme replacement therapy for murine mucopolysaccharidosis type VII J. Clin. Invest. 93 1994 2324 2331
93. P.M. Schlesinger J.S. Rodman T.W. Doebber P.D. Stahl Y.C. Lee C.P. Stowel1 T.B. Kuhlenschmidt The role of extrahepatic tissues in the receptor-mediated plasma clearance of glycoproteins terminated by mannose or N-acetylglucosamine Biochem. J. 192 1980 596 606
94. E.H. Schuchman A.Y. Ioannou M.C. Rattazzi R.J. Desnick Neural gene therapy for inherited diseases with mental retardation: Principles and prospects Ment. Ret. Dev. Disab. Res. Rev. 1 1995 39 48
95. M.E. Schwab K. Suda H. Thoenen Selective retrograde transsynaptic transfer of a protein, tetanus toxin, subsequent to its retrograde axonal transport J. Cell Biol. 82 1979 798 810
96. W.C. Shen H.J.P. Ryser Conjugation of poly-L-lysine to albumin and horseradish peroxidase: A novel method of enhancing the cellular uptake of proteins Proc. Natl. Acad. Sci. (USA) 75 1978 1872 1876
97. W.-C. Shen H.J.-P. Ryser L. Lamanna Disulfide spacer between methotrexate and poly (D-lysine). A probe for exploring the reductive process inendocytosis J. Biol. Chem. 260 1985 10905 10908
98. L. Simpson Pharmacologic experiments on the binding and internalization of the 50,000 dalton carboxyterminus of tetanus toxin at the cholinergic neuromuscular junction J. Pharmacol. Exp. Ther. 234 1985 100 105
99. A. Sofer G. Schwarzmann A.H. Futerman The internalization of short acyl chain analogue of ganglioside GM1 in polarized neurons J. Cell Sci. 109 1996 2111 2119
100. J.M. Staddon K. Herrenknecht C. Schulze C. Smales L.L. Rubin Signal transduction at the blood-brain barrier Biochem. Soc. Trans. 23 1995 475 479
101. J.M. Tager M.N. Hamers A.W. Schram F.A. van den Berg P.J. Rietra C. Loonen J.F. Koster R. Slee An appraisal of human trials in enzyme replacement therapy of genetic diseases R.J. Desnick Enzyme Therapy in Genetic Diseases: 2 1980 A. R. Liss New York 343 359 for the March of Dimes Birth Defect Foundation, Birth Defects: Original Article Series XVI(1)
102. D. Triguero J. Buciak W.M. Pardridge Capillary depletion method for quantification of blood-brain barrier transport of circulating peptides and plasma proteins J. Neurochem. 54 1990 1882 1888
103. B. van Deurs Choroid plexus absorption of horseradish peroxidase from the cerebral ventricles J. Ultrastruct. Res. 55 1976 400 416
104. C. Vogler M. Sands A. Higgins B. Levy J. Grubb E.H. Birkenmeier W.S. Sly Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse Pediatr. Res. 34 1993 837 840
105. C. Vogler M. Sands B. Levy N. Galvin E.H. Birkenmeier W.S. Sly Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: Impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival Pediatr. Res. 39 1996 1050 1054
106. B.U. von Specht B. Geiger R. Arnon J. Passwell G. Keven B. Goldman B.( Padeh Enzyme replacement in Tay-Sachs disease Neurology 29 1979 848 854
107. A.W. Vorbrodt A.S. Lossinsky D.H. Dobrogowska H.M. Wisniewski Cellular mechanisms of the blood-brain barrier (BBB) opening albumin-gold complex Histol. Histopathol. 8 1993 51 61
108. R. Wada C.J. Tifft R.L. Proia Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation Proc. Natl. Acad. Sci. U.S.A. 97 2000 10954 10959
109. S.U. Walkley M.A. Thrall K. Dobrenis P.A. March S. Wurzelmann Bone marrow transplantation in neuronal storage disorders Brain Pathol. 4 1994 376A
110. S.U. Walkley M.A. Thrall K. Dobrenis M. Huang P.A. March D.A. Siegel S. Wurzelmann Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease Proc. Natl. Acad. Sci. (USA) 91 1994 2970 2974
111. S.U. Walkley M.A. Thrall K. Dobrenis Targeting gene products to the brain and neurons using bone marrow transplantation: A cell-mediated delivery system for therapy of inherited metabolic human disease P.R. Lowenstein L.W. Enquist Protocols for Gene Transfer in Neuroscience: Towards Gene Therapy of Neurological Disorders 1996 Wiley London and New York 275 302
112. N.M. Wellhoner Tetanus neurotoxin Rev. Physiol.Biochem. Pharmacol. 93 1982 1 68
113. J.M. Wood Physiology, pharmacology, and dynamics of cerebrospinal fluid J.M. Wood Neurobiology of Cerebrospinal Fluid 1980 Plenum New York 1 15
114. X. Xiao J. Li T.J. McCown R.J. Samulski Gene transfer by adeno-associated virus vectors into the central nervous system Exper. Neurol. 144 1997 113 124
115. E.T. Zhang C.B. Inman R.O. Weller Interrelationships of the pia mater and the perivascular (Virchow-Robin) spaces in the human cerebrum J. Anat. 170 1990 111 123