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Microglia: a cellular vehicle for CNS gene therapy
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by deficiency of the enzyme arylsulfatase A (ARSA). MLD is characterized by progressive demyelination and neurological deficits. Treatment of MLD is still a challenge due to the fact that the blood-brain barrier is a major obst...
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society for Clinical Investigation
2006
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| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1626126/ https://ncbi.nlm.nih.gov/pubmed/17080190 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI30230 |
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