Muscular Dystrophy Cooperative Research Center

肌营养不良症合作研究中心

• 批准号: 7989616
• 负责人: KEVIN P. CAMPBELL
• 金额: $ 149.81万
• 依托单位: UNIVERSITY OF IOWA
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-06-08 至 2015-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7989616
• 关键词: Muscular; Dystrophy; Cooperative; Research; Center

DESCRIPTION (provided by applicant): The overall goal of the University of Iowa MDCRC is to explore therapeutic strategies for the treatment of various muscular dystrophies arising from the abnormal processing of dystroglycan (dystroglycanopathies). The Center will achieve this overall goal by enabling translational research on the dystroglycanopathies and providing advanced diagnostic services. Our MDCRC application is composed of two projects, three cores, and investigators with a proven track record of excellence and collaboration in basic, translational and clinical research. Project 1 will investigate the molecular pathogenesis of the dystroglycanopathies and evaluate various therapeutic strategies, using defined mouse models and dystroglycanopathy patient cells with known mutations. Kevin Campbell will lead this translational project and Steven Moore and Francesco Muntoni will participate in it. A major asset for this project is our recently awarded "GO grant" aimed at the discovery of small molecule effectors of dystroglycan glycosylation. Project 2 will identify and characterize dystroglycanopathy patients and develop infrastructure for therapeutic trials in defined cohorts. Katherine Mathews will be the project leader on this clinical project with participation from Carsten Bonnemann. Core A (Campbell and Moore) is an administrative core and will coordinate the activities within and outside the Center as a means to promote an interactive and collaborative research environment. Core B (Moore), a Muscle-Tissue/Cell-Culture/Diagnostics Core, will serve as a national tissue and cell-culture resource for research, as well as a laboratory for patient diagnostic testing and post-intervention biopsy evaluation for clinical trials. Finally, Core C (Campbell and Mathews) will coordinate our Research Training and Education initiative. Among the support it provides will be a fellowship enabling one medical student per year to perform research in the Center and to participate in the care of patients alongside Katherine Mathews. Thus, the highly integrated cores and projects, coupled with the expanded collaboration with national and international leaders in the field of dystroglycanopathies, will accelerate the tempo of discovery in preclinical translational research, and establish the clinical-trial readiness of a cohort of dystroglycanopathy patients.

描述（由申请人提供）：爱荷华州大学MDCRC的总体目标是探索治疗由肌营养不良蛋白聚糖异常加工引起的各种肌营养不良症（肌营养不良蛋白聚糖病）的治疗策略。该中心将通过对肌营养不良症的转化研究和提供先进的诊断服务来实现这一总体目标。我们的MDCRC应用程序由两个项目，三个核心和研究人员组成，在基础，转化和临床研究方面具有卓越和合作的良好记录。项目1将研究肌营养不良聚糖病的分子发病机制，并评估各种治疗策略，使用定义的小鼠模型和已知突变的肌营养不良聚糖病患者细胞。凯文坎贝尔将领导这个翻译项目，史蒂文摩尔和弗朗西斯科 该项目的一个主要资产是我们最近获得的“GO赠款”，旨在发现肌营养不良聚糖糖基化的小分子效应物。项目2将识别和描述营养不良聚糖病患者，并在确定的队列中开发治疗试验的基础设施。凯瑟琳马修斯将是该临床项目的项目负责人，Carsten Bonnemann将参与该项目。核心A（坎贝尔和摩尔）是一个行政核心，将协调中心内外的活动，作为促进互动和合作研究环境的一种手段。核心B（摩尔），一个肌肉组织/细胞培养/诊断核心，将作为一个国家组织和细胞培养资源的研究，以及一个实验室，病人诊断测试和干预后活检评估的临床试验。最后，核心C（坎贝尔和马修斯）将协调我们的研究培训和教育计划。它提供的支持将包括一项奖学金，使每年一名医科学生能够在该中心进行研究，并与凯瑟琳马修斯一起参与病人的护理。因此，高度整合的核心和项目，加上与国内和国际领导者在肌营养不良症领域的扩大合作，将加快临床前转化研究的发现克里思，并建立肌营养不良症患者队列的临床试验准备。