National Gene Vector Biorepository and Coordinating Center at Indiana University

描述（由申请人提供）：自2008年以来，位于印第安纳州大学的国家基因载体生物库（NGVB）一直是基因治疗社区的国家资源。我们建议继续使用这一资源，并提出以下具体目标： 具体目标1。试剂储存库。NGVB将通过基于网络的基因治疗试剂目录（包括质粒、细胞系和载体）扩大目前在试剂共享方面的努力。在新的资助期内，将特别关注在非病毒基因治疗、IPS细胞生成和转座子技术领域获得的新试剂。 具体目标2。临床前协助：满足FDA临床前药理学和毒理学要求的挑战。NGVB将为药物非临床研究质量管理规范（GLP）样本和已完成基因治疗生物分布和毒性研究的网络数据库（Pharm/Tox数据库）提供存档服务。新举措将包括扩大数据库，以涵盖关键的非GLP研究和建立体外永生化试验。 具体目标3。基因治疗研究的临床试验支持。NGVB将继续提供存档、RCV检测和用于插入位点分析的LAM-PCR，以支持基因治疗临床试验。NGVB还将扩展SeqMap生物信息学工具的功能，该工具有助于分析由桑格或高通量测序技术产生的序列数据。最后，NGVB将通过开发SeqMap 3.0为短读段测序的生物信息学挑战做好准备。 具体目标4。研究和开发用于插入位点分析的合格检测试剂盒。我们建议开发一套标准的试剂和方案，为进行这些研究的研究人员提供重要的内部控制。我们还将开发一种专门设计的标准化检测方法，以满足FDA的要求，并提高检测结果的临床相关性。

DESCRIPTION (provided by applicant): The National Gene Vector Biorepository (NGVB) at Indiana University has serves as a national resource for the gene therapy community since 2008. We propose to continue this resource and propose the following Specific Aims: Specific Aim 1. Reagent Repository. The NGVB will expand current efforts in reagent sharing through a web- based catalogue of gene therapy reagents, including plasmids, cell lines, and vectors. In the new funding period, a special focus will be on obtained novel reagents in the area of non-viral gene therapy, IPS cell generation, and transposon technology. Specific Aim 2. Preclinical Assistance: Meeting the Challenges of FDA Preclinical Pharmacology and Toxicology Requirements. The NGVB will provide archiving services for Good Laboratory Practice (GLP) samples and a web-based database of completed gene therapy biodistribution and toxicity studies (Pharm/Tox database). New initiatives will include an expansion of the database to cover key non-GLP studies and the establishment of the In Vitro Immortalization Assay. Specific Aim 3. Clinical Trial Support of Gene Therapy Studies. The NGVB will continue to provide archiving, RCV testing, and LAM-PCR for insertion site analysis in support of gene therapy clinical trials. The NGVB will also expand the capabilities of the SeqMap bioinformatics tool that assists in the analysis of sequence data generated from Sanger or high-throughput sequencing techniques. Finally, the NGVB will prepare for the bioinformatics challenges of short-read sequencing by developing SeqMap 3.0. Specific Aim 4. Research and Develop a Qualified Assay for Insertion Site Analysis. We propose to develop a standard set of reagents and protocols that will provide important internal controls for investigators conducting these studies. We will also develop a standardize assay designed specifically to meet the FDA requirements and improve the clinical relevance of assay output.