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Article

Safety of Arylsulfatase A Overexpression for Gene Therapy of Metachromatic Leukodystrophy

1 Sept 2007

Abstract excerpt

Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) gene transfer, highlighted a requirement for high levels of arylsulfatase A (ARSA) expression to achieve correction of disease manifestations in the mouse model. Full assessment of the safety of ARSA expression above physiological levels thus...

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PMID: 17845130
DOI: 10.1089/hum.2007.048

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