Core D -Humanized Mouse and Gene Therapy Core

核心D：项目摘要/摘要：加州大学洛杉矶分校-CDU CFAR人源化小鼠和基因的总体目标 治疗(HMGT)核心(核心D)是为需要干细胞的艾滋病毒/艾滋病相关研究提供支持和专业知识 用于体外和体内实验的细胞、基因治疗和人源化小鼠。基因的最新进展 运送载体系统、干细胞技术和动物模型极大地扩展了我们的知识 与病毒发病机制有关的机制，并加速了治疗方法的发展 接近了。新结合的人源化小鼠和基因治疗核心(原人源化小鼠 核心和基因治疗核心)是为了满足日益增长的需求而建立的，以促进和促进基础 以及这些领域的翻译研究，为加州大学洛杉矶分校-CDU CFAR调查员提供服务， 资源、基础设施和专业知识，使这些研究能够以高度整合的 高效、经济实惠的方式。核心将提供高度纯化和良好特性的人CD34 HSPC、胚胎干细胞(HESC)、诱导多能干细胞(IPSC)和慢病毒载体技术 这使得细胞和组织能够进行有效的基因工程来抵御艾滋病毒感染。这些技术是 补充人源化小鼠模型的使用和开发，因为研究人员经常将重点放在 使用新的模型系统来研究人类免疫系统及其操作，以及 HIV感染对人体细胞和体内组织的影响。这为研究人类提供了一个强有力的工具 血细胞发育，研究艾滋病毒感染对人类细胞的影响，并探索保护 人体免疫系统免受艾滋病毒感染。这些技术的使用和人性化小鼠的产生是 高度专业化的程序，由于需要专门的设施、资源和必要的 在这些系统中进行实验的技能和知识，这是核心提供的。核心将提供 为在病毒载体方面经验有限的研究人员，特别是早期研究人员提供咨询 技术、基于干细胞的研究和以老鼠为基础的人性化研究。我们的服务证明是增值的、成本的- 有效地帮助用户利用有限的商业资源或自行进行研究。 来自易接近且知识渊博的核心员工提供的定制技术支持进一步增加了价值 世卫组织可以与调查人员密切合作，排除故障并优化实验，并协助机构 法规遵从性文件。Core D的服务将促进干细胞、基因疗法和 人性化的与老鼠相关的艾滋病毒研究进入治疗应用。

Core D: Project Summary/Abstract: The overall goal of the UCLA-CDU CFAR Humanized Mouse and Gene Therapy (HMGT) Core (Core D) is to provide support and expertise for HIV/AIDS-related research requiring stem cells, gene therapy, and humanized mice for in vitro and in vivo experimentation. Recent advancements in gene delivery vector systems, stem cell technologies and in animal models have significantly expanded our knowledge of the mechanisms associated with viral pathogenesis and have accelerated the development of therapeutic approaches. The newly combined Humanized Mouse and Gene Therapy Core (formerly the Humanized Mouse Core and Gene Therapy Core) was established to meet the increasing demand to promote and facilitate basic and translational research in these areas by providing the UCLA-CDU CFAR investigators with the services, resources, infrastructure, and expertise that enable the performance of these studies in a consolidated, highly efficient, and cost-effective manner. The Core will provide highly purified and well characterized human CD34+ HSPC, embryonic stem cells (hESC), induced pluripotent stem cells (iPSC) and lentiviral vector technologies that enable efficient genetic engineering of cells and tissues to resist HIV infection. These technologies are complementary to the use and development of humanized mouse models, as investigators are often focused on the use of novel model systems with which to study the human immune system, and its manipulation, and the effects of HIV infection on human cells and in tissues in vivo. This provides a powerful tool to examine human blood cell development, to study the effects of HIV infection on human cells, and to explore ways to protect the human immune system from HIV. The use of these technologies and the generation of humanized mice are highly specialized procedures, due to the requirements for specialized facilities, resources, and the necessary skills and knowledge to perform experiments in these systems, which the Core renders. The Core will provide consultation for researchers, in particular to early stage investigators, with limited experience in viral vector technologies, stem cell based, and humanized mouse-based studies. Our services prove a value added, cost- effective approach to users over utilizing limited commercial sources or performing the studies on their own. 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 and assist with institutional regulatory compliance documents. Core D's services will facilitate the translation of stem cell, gene therapy, and humanized mouse-related HIV research into therapeutic applications.