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Busulfan as a Myelosuppressive Agent for Generating Stable High-level Bone Marrow Chimerism in Mice
Published on: April 1, 2015
Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in
Jennifer Conway1, Sarah Dyack, Bruce N A Crooks
1Department of Pediatrics, Division of Genetics, IWK Health Centre, Halifax, Nova Scotia, Canada.
The Journal of Pediatrics
|July 20, 2005
Abstract:
Hurler syndrome is a lysosomal storage disease resulting in fatal cardiac or neurologic sequelae unless alpha-iduronidase production is reconstituted with hematopoietic stem cell transplantation. We report on a 4-year, 6-month-old boy with mixed donor chimerism and low enzyme levels but a normal neurodevelopmental trajectory.
