Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome

Jennifer Conway; Sarah Dyack; Bruce N.A. Crooks; Conrad V. Fernandez

doi:10.1016/j.jpeds.2005.03.005

Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome

Jennifer Conway, Sarah Dyack, Bruce N.A. Crooks, Conrad V. Fernandez

Medicine

Research output: Contribution to journal › Article › peer-review

13 Citations (Scopus)

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.

Original language	English
Pages (from-to)	106-108
Number of pages	3
Journal	Journal of Pediatrics
Volume	147
Issue number	1
DOIs	https://doi.org/10.1016/j.jpeds.2005.03.005
Publication status	Published - Jul 2005

ASJC Scopus Subject Areas

Pediatrics, Perinatology, and Child Health

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Conway, J., Dyack, S., Crooks, B. N. A., & Fernandez, C. V. (2005). Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome. Journal of Pediatrics, 147(1), 106-108. https://doi.org/10.1016/j.jpeds.2005.03.005

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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.",

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AU - Crooks, Bruce N.A.

AU - Fernandez, Conrad V.

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Mixed donor chimerism and low level iduronidase expression may be adequate for neurodevelopmental protection in Hurler syndrome

Abstract

ASJC Scopus Subject Areas

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