Core D -Humanized Mouse and Gene Therapy Core

Core D - 人源化小鼠和基因治疗核心

基本信息

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
项目类别：
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

项目摘要

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：项目总结/摘要：UCLA-CDU CFAR 人源化小鼠和基因的总体目标治疗 (HMGT) 核心（核心 D）是为需要干细胞治疗的艾滋病毒/艾滋病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。基因研究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，并加速了治疗药物的开发接近。新组合的人源化小鼠和基因治疗核心（以前的人源化小鼠核心和基因治疗核心）的建立是为了满足促进和促进基本治疗日益增长的需求通过为 UCLA-CDU CFAR 研究人员提供服务来进行这些领域的转化研究，资源、基础设施和专业知识，使这些研究能够在一个综合的、高度高效且具有成本效益的方式。核心将提供高度纯化且特征明确的人类 CD34+ 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艾滋病毒感染。这些技术是与人源化小鼠模型的使用和开发相辅相成，因为研究人员通常关注使用新颖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。这为检查人类提供了一个强大的工具血细胞发育，研究HIV感染对人体细胞的影响，探索保护血液细胞的方法人体免疫系统免受艾滋病毒侵害。这些技术的使用和人源化小鼠的产生由于需要专门的设施、资源和必要的条件，程序高度专业化在核心渲染的这些系统中进行实验的技能和知识。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、基于干细胞和人性化小鼠的研究。我们的服务证明具有附加值、成本效益避免用户利用有限的商业资源或自行进行研究的有效方法。通过平易近人且知识渊博的核心员工提供的定制技术支持可以增加更多价值他们可以与研究人员密切合作，解决和优化实验并协助机构监管合规文件。 Core D 的服务将促进干细胞、基因治疗和人性化小鼠相关艾滋病毒研究进入治疗应用。