Core--Muscle Biopsy/Cell Culture/ Diagnostics

核心--肌肉活检/细胞培养/诊断

• 批准号: 6960857
• 负责人: STEVEN A MOORE
• 金额: $ 26.41万
• 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: 2005-04-01 至 2010-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6960857
• 关键词: biomedical facility; biopsy; clinical research; cooperative study; human subject; muscle cells; muscular dystrophy; musculoskeletal disorder diagnosis; musculoskeletal disorder therapy; therapy design /development; tissue /cell culture; tissue resource /registry

The Muscle Tissue/Cell Culture/Diagnostics Core for this Wellstone Muscular Dystrophy Cooperative Research Center is a multifaceted laboratory that will be both a local and a national resource for muscular dystrophy research. There are two major goals of the Core. First, the Core will use approximately 4000 existing stored skeletal muscle biopsies to establish a muscle tissue repository. Among the existing muscle biopsies are more than 600 muscular dystrophy samples that include dystrophinopathies (Duchenne and Becker muscular dystrophy), limb-girdle muscular dystrophies, and congenital muscular dystrophies. We will accrue new specimens into the Core from a variety of sources: diagnostic muscle biopsies, therapeutic surgical procedures, endomyocardial biopsies, heart explants, skin biopsies, and autopsies. In addition to these tissues, we will establish fibroblast and myoblast cell lines from diagnostic muscle and skin biopsies. Patients with genetically defined muscular dystrophies will be recruited to undergo biopsies in order to maintain a widely representative spectrum of muscular dystrophy diagnoses. Well characterized tissues and cells from the Core will be made available to research investigators from other centers. The second goal of the Core is to establish a diagnostic resource. In this capacity it will provide diagnostic services that are not readily available through clinical laboratories, it will facilitate development of new diagnostic tests for transfer to the clinical laboratory, and will serve as a post-intervention biopsy evaluation resource for investigators conducting clinical trials. The Core will maintain patient confidentiality, and all testing will be quality controlled to meet CLIA licensing standards. Thus, the Muscle Tissue/Cell Culture/Diagnostics Core will establish a vital resource of muscular dystrophy tissues and cell cultures, provide a critical diagnostic link in expanding the number of patients with specific, molecular diagnoses, and serve as a national biopsy testing resource for clinical trials in muscular dystrophy patients.

Wellstone 肌营养不良症合作研究中心的肌肉组织/细胞培养/诊断核心是一个多方面的实验室，将成为当地和国家肌营养不良症研究的资源。核心有两个主要目标。首先，Core 将使用大约 4000 个现有存储的骨骼肌活检来建立肌肉组织存储库。现有的肌肉活检中有 600 多个肌营养不良症样本，其中包括肌营养不良症（杜氏肌营养不良症和贝克尔肌营养不良症）、肢带型肌营养不良症和先天性肌营养不良症。我们将从各种来源将新样本积累到核心中：诊断性肌肉活检、治疗性外科手术、心内膜心肌活检、心脏外植体、皮肤活检和尸检。此外 对于这些组织，我们将从诊断性肌肉和皮肤活检中建立成纤维细胞和成肌细胞系。将招募患有基因定义的肌营养不良症的患者进行活检，以保持肌营养不良症诊断的广泛代表性。来自核心的经过充分表征的组织和细胞将提供给其他中心的研究人员。核心的第二个目标是建立诊断资源。以此身份，它将提供临床实验室无法提供的诊断服务，将促进新诊断测试的开发以转移到临床实验室，并将作为进行临床试验的研究人员的干预后活检评估资源。核心将保守患者机密，所有测试都将受到质量控制，以满足 CLIA 许可标准。因此，肌肉组织/细胞培养/诊断核心将建立肌营养不良组织和细胞培养的重要资源，为扩大具有特异性分子诊断的患者数量提供关键的诊断环节，并作为肌营养不良患者临床试验的国家活检检测资源。