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Gene therapy of the brain in the dog model of Hurler's syndrome
Abstract
Objective A defect of the lysosomal enzyme alpha-L-iduronidase (IDUA) interrupts the degradation of glycosaminoglycans in mucopolysaccharidosis type I, causing severe neurological manifestations in children with Hurler's syndrome. Delivery of the missing enzyme through stereotactic injection of adeno-associated virus vectors coding for IDUA prevents neuropathology in affected mice. We examined the efficacy and the safety of this approach in enzyme-deficient dogs. Methods Because deficient dogs raise antibodies against IDUA in response to infusion, intracerebral vector injections were combined with an immunosuppressive regimen. Results Treatment was tolerated well. We observed broad dispersion of vector genomes in the brain of efficiently immunosuppressed dogs. The delivery of IDUA to large areas, which could encompass the entire brain, prevented glycosaminoglycan and secondary ganglioside accumulations. This condition was associated with drastic reduction of neuropathology throughout the encephalon. In contrast, vector injection combined with partial immunosuppression was associated with subacute encephalitis, production of antibodies against IDUA in brain tissues, and elimination of genetically modified cells. Interpretation Gene therapy directed to the entire brain is feasible and may be beneficial to children with Hurler's syndrome. The possibility of subacute encephalitis emphasizes the importance of preventing immune response against IDUA, a problem that needs to be cons…
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Various AAV vectors themselves can increase both astrocytic and microglial markers.
“since various AAV vectors themselves can increase both astrocytic and microglial markers (Ciron et al., 2006; Perez et al., 2020)”
From The neuronal retromer can regulate both neuronal and microglial phenotypes of Alzheimer's disease (2022) · cites this paper as Ciron et al., 2006
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Metadata
- Authors
- Ciron, C., Desmaris, N., Colle, M.-A., Raoul, S., Joussemet, B., Vérot, L., Ausseil, J., Froissart, R., Roux, F., Chérel, Y., Ferry, N., Lajat, Y., Schwartz, B., Vanier, M.-T., Maire, I., Tardieu, M., Moullier, P., & Heard, J.-M.
- Venue
- Annals of Neurology
- Publisher
- Wiley
- Published
- 2006-08
- Volume
- 60
- Issue
- 2
- Pages
- 204-213
- ISSN
- 0364-5134
- 1531-8249
- Cited by
- 85
- References
- 0
- Language
- en
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