MOLECULAR AND CELLULAR BIOLOGY OF GENE THERAPY

基因治疗的分子和细胞生物学

• 批准号: 2766098
• 负责人: Bonnie W Ramsey
• 金额: $ 1.7万
• 依托单位: KEYSTONE SYMPOSIA
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-01-01 至 1999-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2766098
• 关键词: cell biology; clinical trials; gene therapy; immunity; meeting /conference /symposium; molecular biology; stem cells; technology /technique development; transfection /expression vector; travel

The field of gene therapy continues to evolve as advances are made in vector development, vector-host cell interactions and understanding of disease pathophysiology. As barriers to successful human gene therapy continue to be addressed at the molecular and cellular level, the research field is progressing towards clinical applications which are beginning to define both the safety of this therapeutic approach and its impact on a variety of diseases. These clinical models are helping investigators define new challenges, including vector production and quality control, in vivo assays to detect gene expression, safety measurements and clinical endpoints. The goal of this conference is to highlight progress in molecular and cellular biology and to establish a basic framework for clinical trial development in human gene therapy. The results of recent clinical trials will be presented. Major barriers to human studies, including limitations of current vectors, host immune response, stem cell biology and disease pathogenesis will be discussed. The meeting will bring together basic scientists and clinical investigators to face these challenges. The program will focus on innovative technologies in vector development, vector-host interactions, challenges of clinical trial development, genetic diseases, stem cell biology, cardiovascular diseases, CNS disorders, AIDS, cancer and vaccine development. Each session will highlight the basic science, preclinical studies and review current clinical trials in progress. The program of speakers emphasizes representation of women and young investigators. The mix of speakers is designed to expose predoctoral and postdoctoral attendees to prominent exponents of each of the major gene therapy methodologies, providing developing scientists the tools for making informed choices in defining their research directions. A portion of the funding sought will be used to defray costs incurred by graduate students and postdoctoral fellows who apply to attend the meeting. Selection of award recipients will be made based on the quality of abstracts submitted for the poster presentation.

随着基因治疗领域的进步，基因治疗领域不​​断发展 载体开发、载体-宿主细胞相互作用和理解 疾病病理生理学。 作为人类基因治疗成功的障碍 继续在分子和细胞水平上进行解决 研究方向正在向临床应用方向发展 开始定义这种治疗方法的安全性及其 对多种疾病的影响。 这些临床模型正在帮助 研究人员确定了新的挑战，包括载体生产和 质量控制、检测基因表达的体内测定、安全性 测量和临床终点。 本次会议的目标是强调分子和 细胞生物学并建立临床试验的基本框架 人类基因治疗的发展。 近期临床结果 将提出试验。 人类研究的主要障碍包括 当前载体的局限性、宿主免疫反应、干细胞生物学 并将讨论疾病发病机制。 会议将带来 基础科学家和临床研究人员一起面对这些 挑战。 该计划将重点关注矢量领域的创新技术 开发、载体-宿主相互作用、临床试验的挑战 发育、遗传疾病、干细胞生物学、心血管 疾病、中枢神经系统疾病、艾滋病、癌症和疫苗开发。每个 会议将重点介绍基础科学、临床前研究和综述 目前临床试验正在进行中。 发言人计划强调妇女和年轻人的代表性 调查人员。 演讲者的组合旨在展示博士生 以及每个专业杰出代表的博士后参加者 基因治疗方法，为发展中的科学家提供工具 在确定研究方向时做出明智的选择。 寻求资金的一部分将用于支付所产生的费用 由申请参加该项目的研究生和博士后研究员 会议。 获奖者的选择将根据 为海报展示提交的摘要的质量。