Determining prognosis and response to treatment for MPS I conditions is currently inadequate, even mutation analysis being imprecise. We hypothesize that neurochemical profiles will differ between MPS I Hurler (post-HCT), Hurler-Scheie (ERT) and age-matched controls and that inflammation and oxidative stress will be more pronounced in ERT patients than post-HCT patients. We hypothesize that within the Hurler-Scheie group, which is more clinically diverse;there will be higher degree of neuroinflammation and oxidative stress in severe patients as defined by genotype and clinical status.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZTR1-CI-8 (01))
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Morris, Jill A
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University of Minnesota Twin Cities
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