Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells

Human Gene Therapy

Volumn 11, Issue 15, 2000, Pages 2117-2127

  Ross, C J D ^a   Bastedo, L ^a   Maier, S A ^a,b   Sands, M S ^c   Chang, P L ^a  

^a MCMASTER UNIVERSITY   (Canada)

^b UNIVERSITY OF ALBERTA   (Canada)

^c WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANIMAL CELL; ARTICLE; COST EFFECTIVENESS ANALYSIS; GENE THERAPY; LYSOSOME STORAGE DISEASE; MICROENCAPSULATION; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; TREATMENT OUTCOME;

ALLELES; ALPHA-GALACTOSIDASE; ANIMALS; ANTIGENS, CD4; BETA-N-ACETYLHEXOSAMINIDASE; CELL LINE; DNA, COMPLEMENTARY; DRUG COMPOUNDING; FIBROBLASTS; GENE THERAPY; GLUCURONIDASE; GLYCOSAMINOGLYCANS; HETEROZYGOTE; HUMANS; KIDNEY; LIVER; MICE; MICE, MUTANT STRAINS; MUCOPOLYSACCHARIDOSIS VII; SPLEEN; TIME FACTORS;

ANIMALIA; MURINAE;

EID: 0034633677     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/104303400750001426     Document Type: Article

References (57)

1. Human gene therapy
2. Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII
3. Gaucher disease as a paradigm of current issues regarding single gene mutations of humans
4. Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency
5. Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation
6. Nonautologous somatic gene therapy
7. Behavioural abnormalities in a murine model of a human lysosomal storage disease
8. Gene transfer and therapy clinical trials - Update
9. Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice
10. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
11. Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer
12. Beta-glucuronidase deficiency mucopolysaccharidosis: Methods for enzymatic diagnosis
13. Large scale purification of phosphorylated recombinant human beta-glucuronidase from overexpressing mouse L cells
14. Long-term functional assessment of encapsulated cells transfected with Tet-On system
15. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
16. Delivery of human factor IX in mice by encapsulated recombinant myoblasts: A novel approach towards allogeneic gene therapy of hemophilia B
17. Retroviral gene transfer into the intestinal epithelium
18. Neuropathology of murine mucopolysaccharidosis type VII
19. Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer
20. AAVant-garde gene therapy
21. Expression of human factor IX by microencapsulated recombinant fibroblasts
22. Protein measurement with the folin phenol reagent
23. The TMC Worldwide Gene Therapy Enrollment Report, end 1996
24. Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells
25. Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts
26. Long-term secretion of therapeutic proteins from genetically modified skeletal muscles
27. The Metabolic basis of inherited disease
28. Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function
29. Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice
30. Delivery of recombinant gene product to canines with nonautologous microencapsulated cells
31. Encapsulation of various recombinant mammalian cell types in different alginate microcapsules
32. Suppression of immunological response against a transgene product delivered from microencapsulated cells
33. Regulation of protein secretion through controlled aggregation in the endoplasmic reticulum
34. Delivery of recombinant gene products to the central nervous system with nonantologous cells in alginate microcapsules
35. Somatic gene therapy of a neurodegenerative lysosomal storage disease using microencapsulated recombinant cells
36. A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII
37. Enzyme replacement therapy for murine mucopolysaccharidosis type VII
38. Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII
39. Humoral immune response limits gene therapy in canine MPS I
40. 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 VII mice
41. Beta glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis
42. Turnover of murine betaglucuronidase. Comparison among liver, kidney, and spleen and between lysosomes and microsomes
43. Central nervous system cell transplantation: A novel therapy for storage diseases?
44. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
45. Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice
46. Cross-correction of beta-glucuronidase deficiency by retroviral vector-mediated gene transfer
47. Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase
48. A murine model of mucopolysaccharidosis VII. Gross and microscopic findings in beta-glucuronidase-deficient mice
49. Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse
50. 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
51. Enzyme replacement in murine mucopolysaccharidosis type VII: Neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy
52. Diagnostic test for mucopolysaccharidosis. I. Direct method for quantifying excessive urinary glycosaminoglycan excretion
53. Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice
54. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic gene transfer
55. Increased susceptibility to the pathogenic effects of wild-type and recombinant herpesviruses in MPS VII mice compared to normal siblings
56. Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation
57. Glycosylphosphatidylinositol-anchored and core protein-intercalated heparan sulfate proteoglycans in rat ovarian granulosa cells have distinct secretory, endocytotic, and intracellular degradative pathways