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

磁共振波谱法确定 MPS I 中的神经炎症和氧化应激 (NESTRASIL)

• 批准号: 8907071
• 负责人: Chester B. Whitley
• 金额: $ 4.56万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-30 至 2017-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8907071
• 关键词: Age; Brain region; Cerebrospinal Fluid; Clinical; Disease; Genotype; Inflammation; Magnetic Resonance Spectroscopy; Measures; Mucopolysaccharidosis I; Mutation Analysis; Oxidative Stress; Patients; Severity of illness; neurochemistry; neuroinflammation; outcome forecast; response

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.

目前正在确定MPS I疾病的预后和治疗反应， 不充分，甚至突变分析也不精确。我们假设神经化学特征会有所不同 MPS I Hurler（HCT后）、Hurler-Scheie（ERT）和年龄匹配的对照组之间的差异， 氧化应激在ERT患者中比HCT后患者中更明显。我们假设， Hurler-Scheie组，临床上更多样化;神经炎症程度更高， 根据基因型和临床状态定义的重症患者的氧化应激。