CORE--GENE THERAPY WORKING GROUP/BIOSTATISTICS

基因治疗工作组和生物统计学核心为基因治疗临床试验的开发，管理和评估提供必要的服务。该核心是一个独特的多学科小组，将基因治疗临床试验的主要参与者聚集在一起，每周举行一次会议。基因治疗工作组和生物统计学核心一直是临床前开发、方案启动、监管批准、方案执行以及数据检索和分析的协调中心。随着患者试验的进行，预计在未来几年内该核心的使用将增加。这个核心为临床前基础科学研究提供了方向，以产生合理设计基因治疗临床试验所需的信息。这导致了在构建逆转录病毒载体和确定干细胞操作的临床前和临床参数方面的合作努力。基因治疗工作组和生物统计学核心的具体服务组成部分是方案制定、法规批准、临床护理与试验研究组成部分的接口以及临床数据管理的协调。这些服务由基因治疗临床研究协调员和基因治疗研究护士实施。基因治疗工作组和生物统计学核心提供了这些复杂举措所需的组织和协调，这些举措需要具有广泛专业知识的个人之间的互动。核心为临床试验的持续分析和质量控制提供了一个环境。这些实用组件的整合代表了临床基因治疗方案的有效，成功开发和执行的基石。

The Gene Therapy Working Group and Biostatistics Core provides the services necessary for the development, administration and evaluation of gene therapy clinical trials. This core is a unique multi-disciplinary group that brings together the principal participants of gene therapy clinical trials in an ongoing weekly meeting. The Gene Therapy Working Group and Biostatistics Core has been the focal point for coordination of preclinical development, protocol inception, regulatory approval, protocol execution, and data retrieval and analysis. The use of this core is expected to increase over the next few years as patient trials are pursued. This core provides direction for preclinical basic science investigations to generate the information necessary to rationally design gene therapy clinical trials. This has resulted in a collaborative effort in construction of retroviral vectors and in defining preclinical and clinical parameters for stem cell manipulation. The specific service component of the Gene Therapy Working Group and Biostatistics Core is coordination of protocol development, regulation approval, interfacing clinical care with research components of the trial, and management of clinical data. These services are implement by the Gene Therapy Clinical Research Coordinator and Gene Therapy Research Nurse. The Gene Therapy Working Group and Biostatistics Core provides the organization and coordination required for these complex initiatives which demand the interaction of individuals with a wide range of expertise performing distinct roles. The core provides an environment for ongoing analysis and quality control of clinical trials. The integration of these practical components represents the cornerstone for efficient, successful development and execution of clinical gene therapy protocols.