Gene and Cellular Therapy Core

项目概述（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、基因传递载体和技术专长的艾滋病毒/艾滋病相关研究提供支持，以便对干细胞进行有效的遗传修饰。基因传递载体系统和干细胞技术的最新进展使人类造血干细胞/祖细胞（HSPC）的基因修饰能够抵抗HIV感染。基因和细胞治疗中心的建立是为了满足日益增长的需求，促进和促进该领域的基础和转化研究，为UCLA CFAR研究人员及其国内外合作者提供高度纯化和充分表征的人类CD34+ 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，使干细胞有效的基因工程，以抵抗艾滋病毒感染。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，特别是早期研究人员提供咨询。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来进行不同类型干细胞的高效基因工程，因此提供这些技术可以显着促进和扩大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。更大的价值来自于可获得的、知识渊博的核心员工提供的定制技术支持，他们可以与研究人员密切合作，排除故障并优化实验，协助制定机构监管合规文件，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干细胞和基因治疗相关的HIV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.