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Article

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

1 Nov 2006

Abstract excerpt

Metachromatic leukodystrophy (MLD) is a demyelinating lysosomal storage disorder for which new treatments are urgently needed. We previously showed that transplantation of gene-corrected hematopoietic stem progenitor cells (HSPCs) in presymptomatic myeloablated MLD mice prevented disease manifestations. Here we show that HSC gene therapy can reverse neurological deficits and neuropathological damage in affected...

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Identifiers

PMID: 17080200
DOI: 10.1172/jci28873

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