Core D -Humanized Mouse and Gene Therapy Core

Core D - 人源化小鼠和基因治疗核心

• 批准号: 10609766
• 负责人: SCOTT G KITCHEN
• 金额: $ 13.98万
• 依托单位: UNIVERSITY OF CALIFORNIA LOS ANGELES
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-04-08 至 2027-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10609766
• 关键词: Acceleration; Acquired Immunodeficiency Syndrome; Animal Model; Animals; Anti-HIV Therapy; Area; Basic Science; Biological Models; Blood Cells; Breeding; CD34 gene; Case Study; Cell Therapy; Cell physiology; Cells; Clinical Trials; Collaborations; Communities; Complex; Consultations; Development; Disease; Embryo; Engraftment; Ensure; Environment; Equipment; Faculty; Gene Delivery; Gene Transduction Agent; Generations; Genes; Genetic; Genetic Engineering; Goals; HIV; HIV Infections; HIV therapy; HIV/AIDS; Hematopoietic; Human; Human Resources; Immune; Immune system; Immunodeficient Mouse; Immunologic Deficiency Syndromes; Immunotherapy; In Vitro; Infrastructure; Institution; International; Knowledge; Lentivirus Vector; Libraries; Mediating; Methods; Modeling; Modification; Mouse Strains; Mus; Operative Surgical Procedures; Pathogenicity; Performance; Play; Predisposition; Preparation; Procedures; Production; Reagent; Records; Research; Research Personnel; Resources; Role; Sendai virus; Services; Site; Source; Speed; System; Technical Expertise; Techniques; Technology; Therapeutic; Tissues; Training; Transduction Gene; Translating; Translational Research; Translations; Viral Pathogenesis; Viral Vector; Work; cell type; clinical translation; cost; cost effective; delivery vehicle; embryonic stem cell; experience; experimental study; gene therapy; genome editing; hematopoietic tissue; hematopoietic tissue transplantation; human stem cells; humanized mouse; implantation; in vivo; in vivo Model; induced pluripotent stem cell; member; mouse model; novel; operation; preclinical development; skills; stem cell gene therapy; stem cell technology; stem cells; therapeutic development; tool; vector

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.

核心D：项目概要/摘要：UCLA-CDU CFAR人源化小鼠和基因工程的总体目标 治疗（HMGT）核心（核心D）是为需要STEM的HIV/AIDS相关研究提供支持和专业知识 细胞、基因治疗和用于体外和体内实验的人源化小鼠。基因组学的最新进展 递送载体系统、干细胞技术和动物模型显着扩展了我们的知识 与病毒发病机制相关的机制，并加速了治疗的发展， 接近。新合并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），以满足日益增长的需求，促进和促进基本的 和翻译研究在这些领域提供UCLA-CDU CFAR调查员的服务， 资源，基础设施和专业知识，使这些研究的性能在一个统一的，高度 高效、经济的方式。核心将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）和慢病毒载体技术 使细胞和组织的有效基因工程能够抵抗HIV感染。这些技术 补充人源化小鼠模型的使用和开发，因为研究人员经常关注 使用新型模型系统来研究人类免疫系统及其操纵，以及 HIV感染对人体细胞和体内组织的影响。这为研究人类 血细胞发育，研究艾滋病毒感染对人体细胞的影响，并探索保护血细胞的方法 人体免疫系统免受艾滋病毒感染。这些技术的使用和人源化小鼠的产生， 高度专业化的程序，由于需要专门的设施，资源和必要的 技能和知识，在这些系统中进行实验，核心呈现。核心将提供 为研究人员提供咨询，特别是对病毒载体经验有限的早期研究人员 技术、基于干细胞的研究和基于人源化小鼠的研究。我们的服务证明了增值，成本- 有效的方法，用户利用有限的商业来源或自己进行研究。 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， 谁可以与研究人员密切合作，以解决和优化实验，并协助机构 法规遵从性文件。Core D的服务将促进干细胞、基因治疗和 人源化小鼠相关的HIV研究转化为治疗应用。