Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

Annals of Neurology

Volumn 48, Issue 1, 2000, Pages 27-38

  Leone, P ^a,c   Janson, C G ^a,c   Bilianuk, L ^b   Wang, Z ^b   Sorgi, F ^d   Huang, L ^d   Matalon, R ^e   Kaul, R ^e   Zeng, Z ^c   Freese, A ^a   McPhee, S W ^f   Mee, E ^g   During, M J ^a,c,f  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^e UNIVERSITY OF TEXAS MEDICAL BRANCH   (United States)

^f UNIVERSITY OF AUCKLAND   (New Zealand)

^g Aucland Hospital   (New Zealand)

Author keywords

[No Author keywords available]

Indexed keywords

AMIDASE; PLASMID DNA; POLYCATION;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CANAVAN DISEASE; CASE REPORT; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; ENZYME ACTIVITY; FETUS; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; IMMUNOCYTOCHEMISTRY; MACACA; MALE; NEWBORN; NONHUMAN; PLASMID; PRIORITY JOURNAL; RAT; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION;

AMIDOHYDROLASES; ANIMALS; ANIMALS, NEWBORN; BETA-GALACTOSIDASE; BRAIN; CANAVAN DISEASE; CELLS, CULTURED; CENTRAL NERVOUS SYSTEM; CHILD, PRESCHOOL; GENE THERAPY; HUMANS; INFANT; MACACA FASCICULARIS; MAGNETIC RESONANCE IMAGING; MALE; PILOT PROJECTS; RATS; RATS, INBRED F344; TREATMENT OUTCOME;

EID: 0033917280     PISSN: 03645134     EISSN: None     Source Type: Journal    
DOI: 10.1002/1531-8249(200007)48:1<27::AID-ANA6>3.0.CO;2-6     Document Type: Article

References (56)

1. Matalon R, Michals K, Sebesta D, et al. Aspartoacylase deficiency and NAA aciduria in patients with Canavan disease. Am J Med Genet 1988;29:463-471
2. Kaul R, Gao GP, Balamurugan K, Matalon R. Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan disease. Nat Genet 1993;5:118-123
3. Leone P, Janson CG, McPhee SJ, During MJ. Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. Curr Opin Mol Ther 1999;1:487-492 [published erratum in Curr Opin Molec Ther 1999;1:658]
4. Leone P, Janson CG, McPhee SJ, During MJ. Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. Curr Opin Mol Ther 1999;1:487-492 [published erratum in Curr Opin Molec Ther 1999;1:658]
5. Baslow M, Suckow RF, Sapirstein V, Hungund BL. Expression of aspartoacylase activity in cultured rat macroglial cells is limited to oligodendrocytes. J Mol Neurosci 1999;13:47-54
6. Ghazizadeh S, Carroll JM, Taichman LB. Repression of retrovirus-mediated transgene expression by interferons: implications for gene therapy. J Virol 1997;71:9163-9169
7. Qin L, Ding Y, Pahud DR, et al. Promoter attenuation in gene therapy: IFN-gamma and TNF-alpha inhibit transgene expression. Hum Gene Ther 1997;17:2019-2029
8. Muruve DA, Barnes MJ, Stillman IE, Libermann TA. Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum Gene Ther 1999;10:965-976
9. Palella TD, Hidaka Y, Silverman LJ, et al. Expression of human HPRT mRNA in brains of mice infected with a recombinant HSV-1 vector. Gene 1989;80:137-144
10. Chiocca EA, Choi BB, Cai WZ, et al. Transfer and expression of the LacZ gene in rat brain neurons mediated by HSV mutants. N Biol 1990;2:739-746
11. Kaplitt MG, Kwong AD, Kleopoulos SP, et al. Expression of a functional foreign gene in adult mammalian brain following in vivo transfer via a HSV-1 defective viral vector. Proc Natl Acad Sci USA 1994;91:8979-8983
12. Fink DJ, Sternberg LR, Weber PC, et al. In vivo expression of beta-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum Gene Ther 1992;3:11-19
13. Ho DY, Mocarski ES, Sapolsky RM. Altering central nervous system physiology with a defective HSV vector expressing the glucose transporter gene. Proc Natl Acad Sci USA 1993;90: 3655-3659
14. During MJ, Naegele JR, O'Malley KL, Geller AI. Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 1994;266:1399-1403
15. Goins WF, Krisky D, Marconi P, et al. HSV vectors for gene transfer to the nervous system. J Neurovirol 1997;3(Suppl 1): S80-S88
16. Lachmann RH, Efstathiou S. Use of HSV-1 for transgene expression within the nervous system. Clin Sci (Colch) 1999;96: 533-541
17. Le Gal La Salle G, Robert JJ, Berrard S, et al. An adenovirus vector for gene transfer into neurons and glia in the brain. Science 1993;259:988-990
18. Davidson BL, Allen ED, Kozarsky KF, et al. A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat Genet 1993;3:219-223
19. Akli S, Caillaud C, Vigne E, et al. Transfer of foreign gene into the brain using adenovirus vectors. Nat Genet 1993;3:224-228
20. Bajocchi G, Feldman SH, Crystal RG, Mastrangeli A. Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors. Nat Genet 1993;3: 229-234
21. Horellou P, Vigne E, Castel MN, et al. Direct intracerebral gene transfer of an adenoviral vector expressing tyrosine hydroxylase in a rat model of Parkinson's disease. Neuroreport 1994; 6:49-53
22. Kaplitt MG, Leone P, Samulski RJ, et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994;8:148-154
23. Peel AL, Zolotukhin S, Schrimsher GW, et al. Efficient transduction of GFP in rat spinal cord neurons using AAV vectors containing cell type-specific promoters. Gene Ther 1997;4: 16-24
24. Freese A, Kaplitt MG, O'Connor WM, et al. Direct gene transfer into human epileptogenic hippocampal tissue with an AAV vector: implications for a gene therapy approach to epilepsy. Epilepsia 1997;38:759-766
25. During MJ, Samulski RJ, Elsworth JD, et al. In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther 1998;5:820-827
26. Elliger SS, Elliger CA, Aguilar CP, et al. Elimination of lysosomal storage in brains of MPSVII mice treated by intrathecal administration of an adeno-associated virus vector. Gene Ther 1999;6:1175-1178
27. Lo WD, Qu G, Sferra TJ, et al. AAV-mediated gene transfer to the brain: duration and modulation of expression. Hum Gene Ther 1999;10:201-213
28. Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996;272:263-269
29. Blomer U, Naldini L, Kafri T, et al. Highly efficient and sustained gene delivery in adult neurons with a lentivirus vector. J Virol 1997;71:6641-6649
30. Suhr ST, Gage FH. Gene therapy in the CNS: the use of recombinant retroviruses. Arch Neurol 1999;56:287-292
31. Muldoon LL, Nilaver G, Kroll RA, et al. Comparison of intracerebral inoculation and osmotic blood-brain barrier disruption for delivery of adenovirus, herpesvirus, and iron oxide particles to normal rat brain. Am J Pathol 1995;147:1840-1851
32. Schwartz B, Benoist C, Abdallah B, et al. Gene transfer by naked DNA into adult mouse brain. Gene Ther 1996;3:405-411
33. Roessler BJ, Davidson BL. Direct plasmid-mediated transfection of adult murine brain cells in vivo using cationic liposomes. Neurosci Lett 1994;167:5-10
34. Iwamoto Y, Yang K, Clifton GL, et al. Liposome-mediated BDNF cDNA transfer in intact and injured rat brain. Neuroreport 1996;7:609-612
35. Zauner W, Brunner S, Buschle M, et al. Differential behaviour of lipid-based and polycation-based gene transfer systems in transfecting primary human fibroblasts: a potential role of polylysine in nuclear transport. Biochim Biophys Acta 1999;1428: 57-67
36. Gao X, Huang L. Potentiation of cationic liposome-mediated gene delivery by polycations. Biochemistry 1996;35:1027-1036
37. Farhood H, Serbina N, Huang L. The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim Biophys Acta 1995;1235:289-295
38. Li S, Huang L. In vivo gene transfer via IV administration of cationic LPD complexes. Gene Ther 1997;4:891-900
39. Li S, Rizzo MA, Bhattacharya S, Huang L. Characterization of cationic LPD complexes for IV gene delivery. Gene Ther 1998; 5:930-937
40. Nabel GJ, Nakel EG, Yang ZY, et al. Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans. Proc Natl Acad Sci USA 1993;90:11307-11311
41. Caplen NJ, Alton EW, Middleton PG, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with CF. Nat Med 1995;1:39-46
42. Li S, Huang L. Lipidic vector systems for gene delivery. Crit Rev Ther Drug Carr Sys 1997;14:173-206
43. Li S, Wu SP, Whitmore M, et al. Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors. Am J Physiol 1999;276(5 Pt 1):L796-L804
44. Philip R, Brunette E, Kilinski L, et al. Efficient and sustained gene expression in primary T lymphocytes and primary and cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cationic liposomes. Mol Cell Biol 1994;14:2411-2418
45. Vieweg J, Boczkowski D, Roberson KM, et al. Efficient gene transfer with adeno-associated virus-based plasmids complexed to cationic liposomes for gene therapy of human prostate cancer. Cancer Res 1995;55:2366-2372
46. Rosenberg GA, Kyner WT, Estrada E. Bulk flow of brain interstitial fluid under normal and hyperosmolar conditions. Am J Physiol 1980;238:F42-F49
47. Bobo RH, Laske DW, Akbasak A, et al. Convection-enhanced delivery of macromolecules to the brain. Proc Natl Acad Sci USA 1994;91:2076-2080
48. Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989;63:3822-3828
49. Kaul R, Casanova J, Johnson AB, et al. Purification, characterization, and localization of aspartoacylase from bovine brain. J Neurochem 1991;56:129-135
50. Paxinos G, Watson C. Atlas of the rat brain in stereotactic coordinates, 4th ed. New York: Academic Press, 1998
51. Kaul R, Gao GP, Matalon R, et al. Identification and expression of eight novel mutations among non-Jewish patients with CD. Am J Hum Genet 1996;59:95-102
52. Traeger EC, Rapin I. The clinical course of CD. Pediatr Neurol 1998;18:207-212
53. Sorgi FL, Bhattacharya S, Huang L. Protamine sulfate enhances lipid-mediated gene transfer. Gene Ther 1997;4:961-968
54. Byrnes AP, Rusby JE, Wood MJ, Charlton HM. Adenovirus gene transfer causes inflammation in the brain. Neuroscience 1995;66:1015-1024
55. Wood MJ, Byrnes AP, Pfaff DW, et al. Inflammatory effects of gene transfer into the CNS with defective HSV-1 vectors. Gene Ther 1994;1:283-291
56. Szarijanni N, Rethelvi M. Differential distribution of small and large neurons in the sacrococcygeal dorsal root ganglia of the cat. Acta Morphol Acad Sci Hung 1979;27:25-35