Gene and Cellular Therapy Core

项目概要（见说明）：基因和细胞治疗核心（CoreG）的总体目标是为需要干细胞、基因递送载体和技术专长的艾滋病毒/艾滋病相关研究提供支持，以便有效地对干细胞进行遗传修饰。基因递送载体系统和干细胞技术的最新进展使得人类造血干/祖细胞（HSPC）的遗传修饰能够抵抗HIV感染。基因和细胞治疗核心的建立是为了满足日益增长的需求，以促进和促进该领域的基础和转化研究，为UCLA CFAR研究人员及其国内和国际合作者提供高度纯化和充分表征的人CD 34 + HSPC，胚胎干细胞（hESC），诱导多能干细胞（iPSC），人类胎儿组织和慢病毒载体技术，使干细胞的有效基因工程，以抵御艾滋病毒感染。核心还为在干细胞和病毒载体技术方面经验有限的研究人员提供咨询，特别是早期研究人员。由于干细胞和载体技术的使用需要专门的专业知识和资源，以有效地对不同类型的干细胞进行基因工程，因此提供这些技术可以大大促进和扩大UCLA CFAR研究活动的范围。我们的服务比利用有限的商业资源更具成本效益。通过可访问和知识渊博的核心工作人员提供的定制技术支持，进一步增加了价值，这些核心工作人员可以与研究人员密切合作，排除故障和优化实验，协助机构监管合规文件，并积极参与干细胞和载体技术的开发和应用。这些核心服务将促进将干细胞和基因治疗相关的艾滋病毒研究转化为治疗应用。

PROJECT SUMMARY (See instructions): The overall goal of the Gene and Cellular Therapy Core (CoreG) is to provide support for HIV/AIDS-related research requiring stem cells, gene delivery vectors and technical expertise for efficient genetic modification of stem cells. Recent advancements in gene delivery vector systems and stem cell technologies have enabled genetic modification of human hematopoietic stem/progenitor cells (HSPC) to resist HIV infection. The Gene and Cellular Therapy Core is established to meet the increasing demand to promote and facilitate basic and translational research in this area by providing UCLA CFAR investigators and their domestic and international collaborators with highly purified and well characterized human CD34+ HSPC, embryonic stem cells (hESC), induced pluripotent stem cells (iPSC), human fetal tissues and lentiviral vector technologies that enable efficient genetic engineering of stem cells to resist HIV infection. The Core also provides consultation for researchers with limited experience in stem cell and viral vector technologies, in particular early stage investigators. As the use of stem cells and vector technology requires specialized expertise and resources for efficient genetic engineering of different types of stem cells, offering access to these technologies can significantly facilitate and expand the scope of UCLA CFAR research activities. Our services are more cost-effective than utilizing the limited commercial sources. Further value is added by customized technical support available from accessible and knowledgeable core staffs who can work closely with investigators to troubleshoot and optimize experiments, assist with institutional regulatory compliance documents and who are actively engaged in development and application of stem cell and vector technologies. These core services will facilitate translation of stem cell and gene therapy-related HIV research into therapeutic applications.