MR Spectroscopy to Determine Neuroinflammation and Oxidative Stress in MPS I (NESTRASIL)

$45,600U54FY2015NSNIH

University Of Minnesota, Minneapolis MN

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Abstract

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.

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