# Xenotransplant and Genome Editing Core

> **NIH NIH U54** · CINCINNATI CHILDRENS HOSP MED CTR · 2022 · $182,703

## Abstract

Abstract
Genetically modified and immunocompromised animals are the gold-standard models for investigating the
molecular regulation of hematopoietic cells in the context of normal development and diseases, and for the
development of novel stem cell-based therapeutic modalities for a wide variety of non-malignant hematological
disorders. The future of hematology research also lies in our ability to examine hematopoietic cell function at
single cell level and correlate single cell behavior with transcriptome and epigenetic profiling. The CCCEH
Xenotransplant and Genome Editing Core (XGEC) is an established, productive and innovative core
designed to provide specialized mouse strains, a wide variety of animal services for efficiency and
standardization of cell transplantation assays, and transgenic animal services in a timely and cost effective
fashion to members of the CCHSCC. The overarching hypothesis is that a centralized core service providing
the myriad mouse needs of CCHSCC members will enhance the effectiveness, timeliness, cost-efficiency and
overall impact of the scientific research and thereby accelerate the discovery and treatment of hematological
diseases.
 XGEC provides specialized mouse strains in a cost effective manner compared with external vendors,
including a collection of immunodeficient mouse strains (i.e., NSG, NSG-3GM3, NRG-SGM3, Rag-/-gc-/-
/WvWv, and B6.BoyJ) utilized for xenotransplantation of human hematopoietic cells, for preclinical studies
concerning gene transfer in humans and mouse hematopoietic stem cells, for human hematopoietic stem cell
and single cell engraftment studies, and for quantitative (competitive) hematopoietic stem cell engraftment
studies (Aim 1). The XGEC further provides specialized animal services allowing for population and single cell
transplantation assays, including animal irradiation and cell transplantation services (Aim 2), and animal
manipulation and monitoring services (Aim 3). In addition, the XGEC offers all services for the generation of
transgenic and genetically–modified mouse lines (Aim 4). Since it began offering CRISPR-Cas services in
March 2014, the core has already generated engineered animal models to CCHMC investigators and among
Centers and within the wider research community. Lastly, XGEC provides technical support and facilitates
research collaborations for comprehensive design and analysis of in vivo studies (Aim 5). These services will
facilitate the sharing of expertise and promote interaction between investigators participating in the CCCEH,
and will attract new core users in the future. Overall, the XGEC offers a unique combination of animal services
and concentrated expertise performed by well-trained personnel under the supervision of Drs. Starczynowski
and Huang in a cost effective and standardized way, thereby providing state-of-the-art investigation of non-
malignant molecular hematology. These services will support the development of new therapeutic modalities
(ph...

## Key facts

- **NIH application ID:** 10458592
- **Project number:** 5U54DK126108-02
- **Recipient organization:** CINCINNATI CHILDRENS HOSP MED CTR
- **Principal Investigator:** Daniel Starczynowski
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $182,703
- **Award type:** 5
- **Project period:** 2021-08-01 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10458592

## Citation

> US National Institutes of Health, RePORTER application 10458592, Xenotransplant and Genome Editing Core (5U54DK126108-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10458592. Licensed CC0.

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