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中文摘要
翻译
病毒载体核心是宾夕法尼亚大学糖尿病研究中心(DRC)成员对使用病毒载体进行基因治疗和基础研究应用感兴趣的重要资源。载体核心提供腺病毒载体、腺相关病毒载体(AAV)和慢病毒载体,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位于该国最重要的基因治疗研究实验室之一,专门生产和分销新型AAV血清型载体和假型慢病毒载体。DRC载体核心的目标如下:利用最先进的设施和专门的专业人员,核心将:(1)为DRC中心成员提供用于糖尿病和相关内分泌紊乱应用的先进载体技术;(2)与DRC执行委员会成员合作,开发专门供DRC成员使用的载体或重组病毒;(3)将宾夕法尼亚大学实验室开发的具有卓越转导特征的新创建的载体过渡到核心,用于验证和分发给DRC成员;(4)就病媒建设和实验设计向刚果民主共和国成员提供咨询;(5)开发一个基于网络的界面,以配合最近开发的病媒核心数据库,使刚果民主共和国成员能够请求服务,监测病媒生产的状态,并获取病媒核心为其实验室生产的所有病媒的历史数据。与更大的宾夕法尼亚州立大学DRC一样,DRC载体核心的总体目标是为宾夕法尼亚大学和周围机构的调查人员提供服务,并帮助推进与糖尿病相关的研究,以实现预防、治疗和治愈这种毁灭性疾病的目标。
英文摘要
The Viral Vector Core is a critical resource for Penn Diabetes Research Center (DRC) members interested in the use of viral vectors for gene therapy and basic research applications. The Vector core provides adenoviral vectors, adeno-associated viral vectors (AAV) and lentiviral vectors and offers a full range of services including cloning, DNA amplification and characterization, vector design and consultation. The Core is located within one of the premier gene therapy research laboratories in the country and specializes in the production and distribution of novel AAV serotype vectors and pseudotyped lentiviral vectors. The objectives of the DRC Vector Core are as follows: Using state-of-the-art facilities and dedicated professional staff, the Core will: (1) provide DRC Center members with access to advanced vector technologies for use in diabetes and related endocrine disorder applications; (2) work with DRC executive committee members to develop vectors or recombinant viruses specifically for use by DRC members; (3) transition newly created vectors with superior transduction profiles developed in Penn laboratories to the Core for validation and distribution to DRC members; (4) provide consultation to DRC members for vector construction and experimental design and (5) develop a web-based interface to accompany the recently developed Vector Core database to enable DRC members to request services, monitor the status of vector production and access historical data on all vectors produced for their laboratory by the Vector Core. The overall goal of the DRC Vector Core, like that of the larger Penn DRC, is to serve investigators at Penn and surrounding institutions, and aid in the advancement of diabetes-related research toward the goal of prevention, treatment, and cures for this devastating disease.
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GENE THERAPY RESOURCE PROGRAM (GTRP): TASK AREA B FOR PRECLINICAL VECTOR CORE LABORATORY SCIENTIFIC ACTIVITIES
  • 批准号:
    10725221
  • 项目类别:
  • 资助金额:
    $79.61万
  • 财政年份:
    2022
  • 负责人:
    JULIE JOHNSTON
  • 依托单位:
GENE THERAPY RESOURCE PROGRAM (GTRP): TASK AREA B FOR PRECLINICAL VECTOR CORE LABORATORY SCIENTIFIC ACTIVITIES
  • 批准号:
    10788012
  • 项目类别:
  • 资助金额:
    $62.81万
  • 财政年份:
    2022
  • 负责人:
    JULIE JOHNSTON
  • 依托单位:
Vector Core
  • 批准号:
    7595332
  • 项目类别:
  • 资助金额:
    $20.43万
  • 财政年份:
    2009
  • 负责人:
    JULIE JOHNSTON
  • 依托单位:
Vector Core
  • 批准号:
    7802299
  • 项目类别:
  • 资助金额:
    $14.0万
  • 财政年份:
    2009
  • 负责人:
    JULIE JOHNSTON
  • 依托单位:
海外基金