Use of nonviral promoters in adenovirus-mediated gene therapy: Reduction of lysosomal storage in the aspartylglucosaminuria mouse

Journal of Gene Medicine

Volumn 8, Issue 6, 2006, Pages 699-706

  Virta, Salli ^a   Rapola, Juhani ^b   Jalanko, Anu ^a   Laine, Minna ^a  

^a NATIONAL PUBLIC HEALTH INSTITUTE   (Finland)

^b UNIVERSITY OF HELSINKI   (Finland)

Author keywords

Adenovirus; Aspartylglucosaminuria; CNS; Lysosomal storage; Mouse model; Nonviral promoter

Indexed keywords

ADENOVIRUS VECTOR; GREEN FLUORESCENT PROTEIN; N4 (BETA N ACETYLGLUCOSAMINYL)ASPARAGINASE; NEURON SPECIFIC ENOLASE; TOLONIUM CHLORIDE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASPARTYLGLYCOSAMINURIA; ASTROCYTE; BRAIN CORTEX; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; CYTOMEGALOVIRUS; DRUG ADMINISTRATION ROUTE; ENZYME ANALYSIS; GENE OVEREXPRESSION; IMMUNOFLUORESCENCE; IMMUNOHISTOCHEMISTRY; LONG TERM CARE; LYSOSOME STORAGE DISEASE; MOUSE; NONHUMAN; PARIETAL LOBE; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; THALAMUS; VIRAL GENE DELIVERY SYSTEM;

ADENOVIRIDAE; ANIMALS; ASPARTYLGLUCOSYLAMINASE; CEREBRAL CORTEX; DISEASE MODELS, ANIMAL; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GENE THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES; LYSOSOMES; MICE; MICE, INBRED C57BL; PROMOTER REGIONS (GENETICS); PROTEIN TRANSPORT; THALAMUS;

ADENOVIRIDAE; ANIMALIA; CYTOMEGALOVIRUS;

EID: 33745281681     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.892     Document Type: Article

References (31)

1. Aula P, Jalanko A, Peltonen L. Aspartylglucosaminuria. In The Metabolic and Molecular Basis of Inherited Disease (8th edn), Scriver CR, Beaudet A, Sly W, Valle D, Childs B, Vogelstein B (eds). McGraw-Hill: New York, 2001; 3535-3550.
2. Saarela J, Laine M, Oinonen C, et al. Molecular pathogenesis of a disease: structural consequences of aspartylglucosaminuria mutations. Hum Mol Genet 2001; 9: 983-995.
3. Saarela J, Oinonen C, Jalanko A, Rouvinen J, Peltonen L. Autoproteolytic activation of human aspartylglucosaminidase. Biochem J 2004; 378: 363-371.
4. Saarela J, Von Schantz C, Peltonen L, Jalanko A. A novel aspartylglucosaminuria mutation affects translocation of aspartylglucosaminidase. Hum Mut 2004; 24: 350-351.
5. Autio S. Aspartylglucosaminuria. Analysis of thirty-four patients. J Ment Defic Res 1972; 1: 1-93.
6. Arvio M, Autio S, Louhiala P. Early clinical symptoms and incidence of aspartylglucosaminuria in Finland. Acta Paediatr 1993; 82: 587-589.
7. Arvio M. Follow-up in patients with aspartylglucosaminuria. I. The course of intellectual functions. Acta Paediatr 1993; 82: 590-594.
8. Kaartinen V, Mononen I, Voncken JW, et al. A mouse model for the human lysosomal disease aspartylglucosaminuria. Nat Med 1996; 2: 1375.
9. Jalanko A, Tenhunen K, McKinney CE, et al. Mice with aspartylglucosaminuria mutation similar to humans replicate the pathophysiology in patients. Hum Mol Genet 1998; 7: 265-272.
10. Tenhunen K, Uusitalo A, Autti T, et al. Monitoring the CNS pathology in aspartylglucosaminuria. J Neuropath Exp Neurol 1998; 57: 1154-1163.
11. Arvio M, Sauna-Aho O, Peippo M. Bone marrow transplantation for aspartylglucosaminuria: follow-up study of transplanted and non-transplanted patients. J Pediatr 2001; 138: 288-290.
12. Malatack JJ, Consolini DM, Bayever E. The status of hematopoietic stem cell transplantation in lysosomal storage disease. Pediatr Neurol 2003; 29: 391-403.
13. Laine M, Richter J, Fahlman C, et al. Correction of peripheral lysosomal accumulation in mice with aspartylglucosaminuria by bone marrow transplantation. Exp Hematol 1999; 27: 1467-1474.
14. Laine M, Ahtiainen L, Rapola J, Richter J, Jalanko A. Bone marrow transplantation in young aspartylglucosaminuria mice: improved clearance of lysosomal storage in brain by using wild type as compared to heterozygote donors. Bone Marrow Transpl 2004; 34: 1001-1003.
15. Kyttälä A, Heinonen O, Peltonen L, Jalanko A. Expression and endocytosis of lysosomal aspartylglucosaminidase in mouse primary neurons. J Neurosci 1998; 18: 7750-7756.
16. Peltola M, Kyttala A, Heinonen O, et al. Decreased lysosomal storage in aspartylglucosaminuria (AGU) mouse brain and liver after adenovirus-mediated gene transfer. Gene Ther 1998; 5: 1314-1321.
17. Akli S, Caillaud C, Vigne E, et al. Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 1993; 3: 224-228.
18. Davidson BL, Allen ED, Kozarsky KF, Wilson JM, Roessler BJ. A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat Genet 1993; 3: 219-223.
19. 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.
20. Harkke S, Laine M, Jalanko A. Aspartylglucosaminidase (AGA) is efficiently produced and endocytosed by glial cells: implication for the therapy of a lysosomal storage disorder. J Gene Med 2003; 5: 472-482.
21. Corish P, Tyler-Smith C. Attenuation of green fluorescent protein half-life in mammalian cells. Protein Eng 1999; 12: 1035-1040.
22. Tollersrud O, Aronson N. Comparison of liver glycoasparaginases from six vertebrates. Biochem J 1992; 282: 891-897.
23. Vogler C, Galvin N, Levy B, et al. Transgene produces massive overexpression of human b-glucuronidase in mice, lysosomal storage of enzyme and strain-dependent tumors. Proc Natl Acad Sci U S A 2003; 100: 2669-2673.
24. Uusitalo A, Tenhunen K, Heinonen O, et al. Towards understanding the neuronal pathogenesis of aspartylglucosaminuria: expression of aspartylglucosaminidase in brain during development. Mol Gen Metab 1999; 67: 294-307.
25. Autti T, Raininko R, Haltia M, et al. Aspartylglucosaminuria: radiologic course of the disease with histopathologic correlation. J Child Neurol 1997; 12: 369-375.
26. Ohashi T, Watabe K, Uehara K, Sly WS, Vogler C, Eto Y. Adenovirus-mediated gene transfer and expression of human β-glucuronidase in the liver, spleen and central nervous system in mucopolysaccharidosis type VII. Proc Natl Acad Sci U S A 1997; 94: 1287-1292.
27. Ghodsi A, Stein C, Derksen T, Yang G, Anderson RD, Davidson BL. Extensive β-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain. Hum Gene Ther 1998; 9: 2331-2340.
28. Stein CS, Ghodsi A, Derksen T, Davidson BL. Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice. J Virol 1999; 73: 3424-3429.
29. Frisella WA, O'Connor LH, Vogler CA, et al. Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Mol Ther 2001; 3: 351-358.
30. Nadeau I, Kamen A. Production of adenovirus vector for gene therapy. Biotechnol Adv 2003; 20: 475-489.
31. Uusitalo A, Tenhunen K, Tenhunen J, Matikainen S, Peltonen L, Jalanko A. Expression and regulation of the human and mouse aspartylglucosaminidase gene. J Biol Chem 1997; 272: 9524-9530.