CORE--GENE THERAPY WORKING GROUP/BIOSTATISTICS

基因治疗工作组和生物统计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。这个核心是一个独特的多学科小组，在持续的每周会议中汇集了基因治疗临床试验的主要参与者。基因治疗工作组和生物统计学核心一直是协调临床前开发、方案制定、监管批准、方案执行以及数据检索和分析的焦点。随着患者试验的进行，这种核心的使用预计将在未来几年内增加。该核心为临床前基础科学研究提供方向，以产生合理设计基因治疗临床试验所需的信息。这促成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核心的具体服务组成部分是协调方案制定，法规批准，将临床护理与试验的研究组成部分连接起来，以及临床数据管理。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核心为这些复杂的倡议提供了组织和协调，这些倡议要求具有广泛专业知识的个人相互作用，发挥不同的作用。核心为临床试验的持续分析和质量控制提供了一个环境。这些实际组成部分的整合是有效、成功地开发和执行临床基因治疗方案的基石。

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.