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: 8934179
• 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: 2015
• 资助国家: 美国
• 起止时间: 2015-08-01 至 2017-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8934179
• 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.

确定预后和对MPS I条件治疗的反应目前是 不足，即使突变分析也不精确。我们假设神经化学特征会有所不同 在MPS I Hurler（HCT后），Hurler-Scheie（ERT）和年龄匹配的对照之间以及炎症和 与HCT后患者相比，ERT患者的氧化应激将更明显。我们假设这内 Hurler-Scheie Group在临床上更多样化；神经炎症的程度更高 由基因型和临床状况定义的严重患者的氧化应激。