# Core D -Humanized Mouse and Gene Therapy Core

> **NIH NIH P30** · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2023 · $139,757

## Abstract

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.

## Key facts

- **NIH application ID:** 10609766
- **Project number:** 5P30AI152501-02
- **Recipient organization:** UNIVERSITY OF CALIFORNIA LOS ANGELES
- **Principal Investigator:** SCOTT G KITCHEN
- **Activity code:** P30 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $139,757
- **Award type:** 5
- **Project period:** 2022-04-08 → 2027-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10609766

## Citation

> US National Institutes of Health, RePORTER application 10609766, Core D -Humanized Mouse and Gene Therapy Core (5P30AI152501-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10609766. Licensed CC0.

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