Core D -Humanized Mouse and Gene Therapy Core

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.