# Xenograft Core

> **NIH NIH P01** · OREGON HEALTH & SCIENCE UNIVERSITY · 2020 · $238,668

## Abstract

Project Summary
The evaluation of candidate therapeutic agents for the treatment of bone marrow failure in
patients with Fanconi anemia (FA) is constrained by several challenges including the disease
heterogeneity and the limited number of primary human hematopoietic cells available to study.
In vitro assays of human hematopoiesis do not provide information about the correction of long-
term multilineage hematopoiesis, a critical endpoint that determines the efficacy of therapy for
bone marrow failure in FA and related disorders. The predictive value of preclinical studies can
be significantly enhanced by the use of robust in vivo modelling. We have developed a novel,
double-chimeric xenograft model that allows us for the first time to rigorously test candidate
therapeutic agents for their potential to correct steady-state human FA deficient hematopoiesis.
The goal of this Xenograft Core is to fully evaluate the acute and chronic effects of the
administration of lead therapeutic compounds identified in Projects 1 & 2 of this proposal.

## Key facts

- **NIH application ID:** 9983794
- **Project number:** 5P01HL048546-25
- **Recipient organization:** OREGON HEALTH & SCIENCE UNIVERSITY
- **Principal Investigator:** WILLIAM H FLEMING
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $238,668
- **Award type:** 5
- **Project period:** — → 2022-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9983794

## Citation

> US National Institutes of Health, RePORTER application 9983794, Xenograft Core (5P01HL048546-25). Retrieved via AI Analytics 2026-06-10 from https://api.ai-analytics.org/grant/nih/9983794. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*