From gene transfer to gene therapy in lysosomal storage diseases affecting the central nervous system

Annals of Medicine

Volumn 33, Issue 1, 2001, Pages 28-36

  Poenaru, L ^a  

^a CHU Cochin Port Royal ^*  (France)

Author keywords

Central nervous system; Gene therapy; Lysosomal diseases; Mucopolysaccharidoses; Neurolipidoses

Indexed keywords

GENE THERAPY; GENE TRANSFER; HUMAN; IMMUNOGENICITY; LYSOSOME; LYSOSOME STORAGE DISEASE; PRIORITY JOURNAL; REVIEW; TREATMENT OUTCOME;

EID: 0035069561     PISSN: 07853890     EISSN: None     Source Type: Journal    
DOI: 10.3109/07853890109002057     Document Type: Review

References (89)

1. Lysosomal storage diseases
2. Alpha galactosidase A deficiency: Fabry disease
3. Glycogen storage disease type II: Acid alpha glucosidase (acid maltase) deficiency
4. The GM2 gangliosidoses
5. Batten's disease: Eight genes and still counting?
6. Neuropsychiatric aspects of the adult variant of Tay-Sachs disease
7. First trimester prenatal diagnosis of metabolic diseases: A survey in countries from the European community
8. Prevention of lysosomal storage in Tay-Sachs mice treated with N-Butyldeoxynojirimycin
9. Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin
10. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
11. Enzyme therapy in Gaucher disease type I: Dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months
12. Pathological findings in Gaucher disease type II patients following enzyme therapy
13. Enzyme infusion therapy of the Norrbottnian (type 3) Gaucher disease
14. Prospective study of neurological responses to treatment with macrophage-targeted glucocerebrosidase in patients with type III Gaucher's disease
15. Allogeneic bone marrow transplantation for lysosomal storage diseases
16. Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation
17. Bone marrow transplantation for mucopolysaccharidosis type 1: Experience of two British centers
18. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy
19. Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice
20. Particularities of lysosomal storage diseases gene therapy
21. Immune parameters affecting adenoviral vector gene therapy in the brain
22. Immune responses to adenovirus and adeno-associated virus in humans
23. Gene transfer by naked DNA into adult mouse brain
24. Gene therapy using HVJ-liposomes: The best of both worlds?
25. A new adenoviral vector: Replacement of all viral coding sequences with 28kb of DNA independently expressing both full-length dystrophin and beta-galactosidase
26. Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis
27. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene
28. Peripheral infection with adenovirus causes unexpected long-term brain inflammation in animals injected intracranially with first-generation, but not with high-capacity adenovirus vectors: Towards realistic long-term neurological gene therapy for chronic diseases
29. Selective and rapid uptake of adeno-associated virus type 2 in brain
30. Restoration of arylsulfatase A activity in human-metachromatic-leucodystrophy fibroblasts via retroviral-vector-mediated gene transfer
31. Expression of the human glucocerebrosidase and arylsulfatase A genes in murine and patient primary fibroblasts transduced by an adeno-associated virus vector
32. HSV as a gene transfer vector for the nervous system
33. Deletion of multiple immediate-early genes from herpes simplex virus reduces cytotoxicity and permits long-term gene expression in neurons
34. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
35. HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells
36. Gene transfer to the nigrostriatal system by hybrid herpes simplex virus/adeno-associated virus amplicon vectors
37. Adenoviral vector as a gene delivery system into cultured rat neuronal and glial cells
38. An adenovirus vector for gene transfer into neurons and glia in the brain
39. Restoration of hexosaminidase A activity in human Tay-Sachs fibroblasts via adenoviral vector-mediated gene transfer
40. 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
41. Neuropathology of mice with targeted disruption of hexa gene, a model of Tay-Sachs disease
42. Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency
43. Model mice and human disease
44. Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism
45. Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases
46. Apoptotic cell death in mouse models of GM2 gangliosidosis and observation on human Tay Sachs and Sandhoff diseases
47. Gene delivery into the brain using virus vectors
48. A model system for in vivo gene transfer into the central nervous system using an adenoviral vector
49. Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors
50. Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors
51. Direct intracranial injection of recombinant adeno-associated virus at birth corrects lysosomal storage and improves cognitive function in the mucopolysaccharidosis VII mouse
52. Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector
53. Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a non-pathogenic herpes simplex virus type 1 vector
54. Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer
55. Systemic hyperosmolality improves β-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer
56. Sustained production of beta-glucuronidase from localised 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
57. Microtubule-mediated transport of incoming herpes simplex virus 1 capsids to the nucleus
58. Retrograde axonal transport of herpes simplex virus: Evidence for a single mechanism and a role for tegument
59. Transfer of foreign genes into the brain using adenovirus vectors
60. Gene delivery into the central nervous system by nasal instillation in rats
61. Absence of an immune response in the spinal cord with retrograde transport of an adenoviral vector
62. Estimation of blood-brain barrier crossing of drugs using molecular size and shape, and H-bonding descriptors
63. In vivo protein transduction: Delivery of a biologically active protein into the mouse
64. Gene therapy in the CNS
65. Multipotent neural cell lines can engraft and participate in development of mouse cerebellum
66. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain
67. Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells
68. Neural stem cells for the treatment of the twitcher mouse model of Krabbe disease
69. Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules
70. Marrow stromal cells migrate throughout forebrain and cerebellum, and they differentiate into astrocytes after injection into neonatal mouse brains
71. Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically-modified skin fibroblasts
72. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
73. Adenoviral gene therapy of the Tay-Sachs disease in hexosaminidase A-deficient knock-out mice
74. 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
75. Peroral gene therapy of lactose intolerance using an adeno-associated virus vector
76. In utero gene therapy
77. Adenovirus-mediated gene transfer to the peritoneum and hepatic parenchyma of fetal mice in utero
78. Ultrasonographically guided direct gene transfer in utero: Successful induction of beta-galactosidase in a rabbit model
79. Successful expression of beta-galactosidase and factor IX transgenes in fetal and neonatal sheep after ultrasound-guided percutaneous adenovirus vector administration into the umbilical vein
80. Adenovirus-mediated gene transfer during initial organogenesis in the mammalian embryo is promoter-dependent and tissue-specific
81. In utero gene therapy: Transfer and long-term expression of the bacterial neo gene in sheep after direct injection of retrovital vectors into preimmune fetuses
82. Gene therapy for canine alpha-L-iduronidase deficiency: In utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease
83. Human fetal gene therapy: Moral and ethical questions
84. Prospects for in utero human gene therapy
85. Gene transfer approaches to the lysosomal storage disorders
86. Continuous erythropoietin delivery by muscle-targeted gene transfer using in vivo electroporation
87. FDA halts all gene therapy trials at Penn
88. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides
89. Correction of the UDP-glucuronosyltransferase gene defect in the Gunn rat model of Crigler-Najjar syndrome type I with a chimeric oligonucleotide