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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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| Autori principali: | , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2012
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| Soggetti: | |
| Accesso online: | 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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