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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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Autor principal: Neumann, Harald
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2006
Matèries:
Accés en línia: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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