National Gene Vector Biorespository and Coordinating Center at Indiana University

描述（由申请人提供）：在本申请中，我们提出了一个综合性的国家基因载体库（NGVB），其满足RFA-RR-07- 002中所述的所有目标。NGVB的具体目标是：具体目标1。开发和监督基因治疗试剂库。NGVB将收集各种各样的基因治疗试剂，供学术研究人员进行临床前研究。具体目标2。存档临床试验或药理学/毒理学研究中采集的样本。NGVB将寻求为必须收集的生物标本提供安全可靠的储存场所，以保持符合FDA或其他监管机构的要求。具体目标3。药理学/毒理学数据库的扩展。印第安纳州大学维护了一个基于网络的药理学/毒理学数据库，允许研究人员搜索与其研究领域相关的研究。该独特的资源将成为NGVB数据库的模型，并允许研究人员查看已在FDA存档的研究设计。具体目标4。作为协调中心。NGVB协调中心将促进具体目标1-3内的活动。此外，它将（1）组织一个外部咨询委员会;（2）制定一个政策和程序手册，确定NGVB的活动;（3）开展外联活动;（4）为寻求基因治疗试剂的研究人员提供“服务台”功能;（5）主持一个新的和独特的生物信息学工具，帮助进行载体插入位点分析，SeqMap。具体目标5.插入突变的研究。为了满足RFA的研究要求，我们将建立在我们正在进行的小鼠基因治疗模型的基础上，并将慢病毒载体插入模式与我们现有的逆转录病毒插入数据库进行比较。如果这个系统被证明是克隆选择的预测器，它可以用来验证其他的，理想的体外风险模型。

DESCRIPTION (provided by applicant): In this application, we propose a comprehensive National Gene Vector Repository (NGVB) which meets all the goals stated in RFA-RR-07-002.The Specific Aims of the NGVB are: Specific Aim 1. Develop and Oversee a Repository of Gene Therapy Reagents. The NGVB will gather a wide variety of gene therapy reagents that will be made available to academic investigators for preclinical investigation. Specific Aim 2. Archive Samples Collected in the Performance of Clinical Trials or Pharmacology/Toxicology Studies. The NGVB will seek to provide a safe and secure storage site for biospecimens that must be collected to maintain compliance with FDA or other regulatory agencies. Specific Aim 3. Expansion of a Pharmacology/Toxicology Database. Indiana University has maintained a web-based Pharmacology/Toxicology Database that allows investigators to search for studies that are relevant to their area of research. The unique resource will become the model for NGVB database and allow investigators to view the design of studies already on file with the FDA. Specific Aim 4. Serve as a Coordinating Center. The NGVB Coordinating Center will facilitate the activities within Specific Aims 1-3. In addition, it will (1) organize an External Advisory Boards; (2) develop a Policy and Procedure Manual that defines the activities of the NGVB; (3) conduct outreach activities; (4) provide a "help-desk" function for investigators seeking gene therapy reagents; (5) host a new and unique bioinformatics tool that aids in vector insertion site analysis, SeqMap. Specific Aim 5. Research on Insertional Mutagenesis. To address the research requirements of the RFA, we will build on our ongoing work in a murine gene therapy model and will compare the lentiviral vector insertion pattern with our existing database of retroviral insertions. If this system proves to be a predictor of clonal selection, it can be used to validate other, ideally in vitro, models of risk.