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Retroviral Vector-mediated Gene Therapy to Mucopolysaccharidosis I Mice Improves Sensorimotor Impairments and Other Behavior Deficits
Mucopolysaccharidosis I (MPS I) is a lysosomal storage disease due to α-L-iduronidase (IDUA) deficiency that results in the accumulation of glycosaminoglycans (GAG). Systemic gene therapy to MPS I mice can reduce lysosomal storage in the brain, but few data are available regarding the effect upon be...
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| Auteurs principaux: | , , , , , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2012
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3548941/ https://ncbi.nlm.nih.gov/pubmed/22983812 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-012-9530-x |
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