Vector Core

病毒载体核心是宾夕法尼亚大学（UPenn）内外研究人员的重要资源，他们对使用病毒载体进行基因治疗进行临床前研究感兴趣，并有可能转化为临床。Vector Core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扩增和表征、载体设计和咨询。核心在生产基础研究载体方面拥有十多年的经验，并与该国首屈一指的基因治疗实验室之一密切相关。位于宾夕法尼亚大学校园的最先进的设施，核心配备了资源，设备和专业人员的敬业精神，以实现本申请中描述的目标和目的。 核心专门生产和销售在美国开发的新型AAV血清型载体。 Wilson实验室，包括AAVS，该POI的临床候选血清型。的一个组成部分 核心是其质量控制程序，由独立的质量控制组执行。虽然 Core生产的载体是研究级的，已经开发了质量控制测定， 与临床级材料和分级放行标准相关， 释放设施中产生的任何载体。核心的总体目标是为参与者提供 该POI具有先进的载体技术，用于临床前研究，重点是基因开发 鸟氨酸转氨甲酰酶缺乏症（OTCD）的治疗。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）进行稳健的质量管理 控制每种载体，并遵守为每种载体平台建立的放行标准，以及（3） 根据项目的需要进行工艺和分析的开发。 相关性（参见说明）： 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 利用病毒载体研究疾病机制，开发基因疗法， 代谢疾病，包括尿素循环障碍。

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.