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: 10458373
• 负责人: SCOTT G KITCHEN
• 金额: $ 13.83万
• 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/10458373
• 关键词: AIDS/HIV problem; 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; Custom; Development; Disease; Embryo; Engraftment; Ensure; Environment; Equipment; Facility Accesses; Faculty; Gene Delivery; Gene Transduction Agent; Generations; Genes; Genetic; Genetic Engineering; Goals; HIV; HIV Infections; Hematopoietic; Human; Human Resources; Immune; Immune system; Immunodeficient Mouse; Immunotherapy; In Vitro; Infrastructure; Institution; International; Knowledge; Lentivirus Vector; Libraries; Mediating; Methods; Modeling; Modification; Mouse Strains; Mus; Operative Surgical Procedures; Pathogenicity; Performance; Play; 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; base; 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：项目摘要/摘要：UCLA-CDU CFAR人源化鼠标和基因的总体目标 治疗（HMGT）核心（核心D）是为与艾滋病毒/艾滋病相关的研究提供支持和专业知识，需要STEM 细胞，基因治疗和人源化小鼠，用于体外和体内实验。基因的最新进展 输送矢量系统，干细胞技术和动物模型已大大扩展了我们的知识 与病毒发病机理相关的机制，并加速了治疗的发展 方法。新组合的人源性小鼠和基因疗法核心（以前是人源化的小鼠 建立了核心和基因疗法核心），以满足促进和促进基本的日益增长的需求 以及这些领域的转化研究，通过为UCLA-CDU CFAR调查人员提供服务， 资源，基础架构和专业知识，可以在合并的，高度 高效且具有成本效益的方式。核心将提供高度纯净且特征良好的人CD34+ HSPC，胚胎干细胞（HESC），诱导多能干细胞（IPSC）和慢病毒载体技术 这使细胞和组织的有效基因工程能够抵抗HIV感染。这些技术是 互补的人性化小鼠模型的使用和开发，因为研究人员通常专注于 使用新型模型系统来研究人类免疫系统及其操纵以及 艾滋病毒感染对人体细胞和体内组织中的影响。这提供了一个强大的工具来检查人类 血细胞发育，研究HIV感染对人类细胞的影响，并探索保护方法 艾滋病毒的人类免疫系统。这些技术的使用和人性化的小鼠的产生是 高度专业化的程序，由于对专业设施，资源和必要的要求 在这些系统中执行实验的技能和知识，这是核心提供的。核心将提供 咨询研究人员，尤其是早期研究人员的咨询，在病毒媒介方面经验有限 技术，基于干细胞的技术和人源化的研究。我们的服务证明了一个增值，成本 - 使用有限的商业资源或自己进行研究的有效方法。 通过可访问和知识渊博的核心员工获得的定制技术支持添加了进一步的价值 谁可以与调查人员紧密合作，以进行故障排除和优化实验并协助机构 法规合规文件。核心D的服务将促进干细胞，基因治疗和 人源化小鼠相关的HIV研究对治疗应用。