Muscular Dystrophy Cooperative Research Center

肌营养不良症合作研究中心

• 批准号: 7074057
• 负责人: KEVIN P. CAMPBELL
• 金额: $ 143.04万
• 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 2010-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7074057
• 关键词: clinical research; cooperative study; muscular dystrophy; musculoskeletal disorder therapy; therapy design /development

Description (provided by applicant): Muscular dystrophies are a diverse group of inherited disorders characterized by progressive muscle weakness and wasting. The overall goal of this Muscular Dystrophy Cooperative Research Center is to explore therapeutic strategies for the treatment of various muscular dystrophies. The Center will achieve this overall goal by enabling translational research on muscular dystrophies and providing advanced diagnostic services. The MDCRC is composed of three research projects, three cores and investigators with a proven track record of excellence and collaboration. The Director and Co-director, Kevin Campbell and Steven Moore, are investigators with established records in basic, translational and clinical research on muscular dystrophy. Project 1 (Campbell and Barresi) will use mouse models to explore the therapeutic potential of improving muscle membrane maintenance and repair for the treatment of Duchenne muscular dystrophy. Project 2 (Mathews, Campbell, Weiss and Romitti) will study muscular dystrophy patients with fukutin related protein mutations and develop mouse models in order to understand the pathogenesis of this disease and possible therapeutic strategies. Project 3 (Yang, Williamson and Barresi) will study the development of embryonic stem cells as therapeutic tools for stem cell treatments. Core A (Campbell and Moore) is an administrative core which will coordinate the activities within and outside the Center and will promote an interactive and collaborative research environment. Core B (Moore and Barresi) is a Muscle Tissue/Cell Culture/Diagnostics Core that will serve as a national tissue and cell culture resource for research as well as a laboratory for patient diagnostic and post-intervention biopsy evaluation for clinical trials. Finally, Core C (Williamson and Yang), the Human ES Cell Targeting Core, will use gene targeting strategies to produce ES cell lines with specific muscular dystrophy mutations, and thus serve as a national resource of targeted stem cells.

描述（由申请人提供）：肌肉萎缩症是一组以进行性肌肉无力和萎缩为特征的遗传性疾病。这个肌肉萎缩症合作研究中心的总体目标是探索治疗各种肌肉萎缩症的治疗策略。该中心将通过实现肌肉萎缩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。MDCRC由三个研究项目、三个核心和具有卓越和合作记录的研究人员组成。主任和联合主任Kevin Campbell和Steven Moore是在肌肉萎缩症的基础、转化和临床研究方面有着既定记录的研究人员。项目1 （Campbell和Barresi）将使用小鼠模型来探索改善肌膜维持和修复治疗杜氏肌营养不良的治疗潜力。项目2 （Mathews, Campbell， Weiss和Romitti）将研究伴有fukutin相关蛋白突变的肌营养不良患者，并建立小鼠模型，以了解这种疾病的发病机制和可能的治疗策略。项目3 （Yang， Williamson和Barresi）将研究胚胎干细胞作为干细胞治疗的治疗工具的发展。核心A （Campbell和Moore）是一个行政核心，将协调中心内外的活动，并将促进互动和合作的研究环境。Core B （Moore and Barresi）是一个肌肉组织/细胞培养/诊断核心，将作为国家组织和细胞培养研究资源，以及用于患者诊断和临床试验干预后活检评估的实验室。最后，核心C (Williamson和Yang)，即人类胚胎干细胞靶向核心（Human ES Cell Targeting Core），将利用基因靶向策略生产具有特定肌肉萎缩症突变的胚胎干细胞系，从而成为靶向干细胞的国家资源。