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Vector Core

矢量核心

基本信息

批准号：
8326903
负责人：
JULIE JOHNSTON
金额：
$ 11.57万
依托单位：
UNIVERSITY OF PENNSYLVANIA
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/8326903
关键词：
Adenovirus Vector Basic Science Biological Assay Clinic Clinical Cloning Consultations Contracts Control Groups Country DNA amplification Development Disease Equipment Evaluation Gene Delivery Generations Grant Guidelines Hereditary Disease Instruction Laboratories Lentivirus Vector Liver Measures Metabolic Diseases Ornithine carbamoyltransferase deficiency Participant Pennsylvania Phase Process Production Quality Control Regulation Research Research Personnel Resources Serotyping Services Technology Translating Translations Universities Viral Viral Vector adeno-associated viral vector assay development base design disease mechanisms study experience gene therapy gene transfer vector good laboratory practice interest meetings novel pre-clinical pre-clinical research preclinical study programs therapeutic development urea cycle vector

项目摘要

The Viral Vector Core is a critical resource for investigators both within and outside the University of Pennsylvania (UPenn) interested in the use of viral vectors for gene therapy for preclinical research with the potential to translate into the clinic. The Vector Core provides adenoviral vectors, lentiviral vectors and adeno-associated viral vectors (AAV) and offers a full range of services including cloning, DNA amplification and characterization, vector design and consultation. The Core has over a decade of experience in the production of vectors for basic research and is closely associated with one of the premier gene therapy laboratories in the country. Located in a state-of-the art facility on the UPenn campus, the Core is fully equipped with the resources, equipment and dedicated staff of professionals to carry out the objectives and aims described in this application. The Core specializes in the production and distribution of novel AAV serotype vectors developed in the Wilson laboratory at Penn, including AAVS, the Clinical Candidate serotype forthis POI. An integral part of the Core is its quality control program which is carried out by a separate Quality Control Group. Although the vectors produced by the Core are research grade, quality control assays have been developed that are relevant to clinical grade material and graded release criteria are in place which must be met before the release of any vector produced in the facility. The overally objective of the Core is to provide the participants of this POI with advanced vector technologies for preclinical studies focusing on the development of gene therapy for ornithine transcarbamylase deficiency (OTCD). The Specific Aims are as follows: (1) to produce high quality preclinical vector designed according to the needs of each project; (2) to conduct robust quality control on each vector and adhere to the release criteria established for each vector platform and (3) to conduct process and assay development relevant to the needs ofthe program project. RELEVANCE (See instructions): The Viral Vector Core provides advanced vector technologies to investigators interested in gene delivery using viral-based vectors to study disease mechanisms and develop gene therapies for genetic and metabolic diseases including urea cycle disorders.

对于宾夕法尼亚大学(UPenn)内外的研究人员来说，病毒载体核心是一个关键资源，他们对将病毒载体用于基因治疗的临床前研究感兴趣，并有可能转化为临床。载体核心提供腺病毒载体、慢病毒载体和腺相关病毒载体(AAV)，并提供全方位的服务，包括克隆、DNA扩增和鉴定、载体设计和咨询。Core在基础研究载体的生产方面拥有十多年的经验，并与该国一流的基因治疗实验室密切相关。Core位于宾夕法尼亚大学校区最先进的设施中，配备齐全的资源、设备和敬业的专业人员，以实现本申请中描述的目标和目的。科尔公司专门生产和销售新型AAV血清型载体。宾夕法尼亚大学的威尔逊实验室，包括AAVs，这种POI的临床候选血清型。一个不可分割的部分核心是由一个单独的质量控制组执行的质量控制程序。尽管核心产生的载体是研究级的，已经开发出质量控制分析，这些分析是与临床等级相关的材料和分级释放标准已到位，必须在释放在该设施中产生的任何媒介。核心的总体目标是为参与者提供利用先进的载体技术进行临床前研究，重点是基因的开发治疗鸟氨酸转氨酶缺乏症(OTCD)。具体目标如下：(1)生产根据每个项目的需要设计高质量的临床前载体；(2)进行稳健的质量控制每个载体，并遵守为每个载体平台建立的发布标准，以及(3) 根据计划项目的需要进行相关的过程和分析开发。相关性(请参阅说明)：病毒载体核心为对基因传递感兴趣的研究人员提供先进的载体技术利用基于病毒的载体研究疾病机制并开发基因治疗代谢性疾病，包括尿素循环障碍。