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Article

Microglia: a cellular vehicle for CNS gene therapy

1 Nov 2006

Abstract excerpt

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 obstacle for most therapeutic substances. In this issue of the JCI, Biffi et al. report that genetically modified...

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Share to Adenosine and Purinergic Signaling
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PMID: 17080190
DOI: 10.1172/jci30230

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