# Preclinical Mouse Model Core

> **NIH NIH U19** · JACKSON LABORATORY · 2024 · $658,430

## Abstract

PROJECT SUMMARY PRECLINICAL MOUSE MODEL CORE
Mouse models are a critical component to both understanding disease mechanisms and to serve as a key
platform for preclinical testing of novel therapeutics. Although in vitro systems are very useful for screening a
large number of gene editing designs, mice provide a tractable, mammalian system to clearly demonstrate
functional efficacy in vivo using a validated model of disease. These data are critical to support an IND for new
treatments. The Preclinical Mouse Model Core is structured to provide centralized and scaled access to models,
in vivo delivery expertise, and phenotyping capabilities to support the individual disease research projects for
this program, towards our goal of IND-enabling validation of genomic editing therapeutics. The Jackson
Laboratory (JAX) houses the world's largest collection of mouse models of human disease, including all key
models for this program. In addition, JAX has leveraged its expertise and scale to develop a broad set of services
that will serve to further support the needs of the individual projects in a cost-effective and high-throughput
manner. This includes our Genetic Engineering Technology team who has, over the past 3 years, executed more
than 2,200 CRISPR/Cas9-based projects of all levels of complexity for 400+ investigators around the world,
including the large-scale, high-throughput KOMP2 Center that has produced more than 1,000 gene knockouts.
JAX has also built, outfitted, and opened a world-class mouse phenotyping clinic, the JAX Center for Biometric
Analysis, which serves both individual faculty projects as well as larger-scale phenotyping needs. Critically, our
proposed Core will build on our current experience as a Small Animal Testing Center for the current Phase of
the Somatic Cell Genome Editing consortium. This program leverages the capabilities of the JAX In Vivo
Pharmacology Service, which provides comprehensive pre-clinical efficacy, tolerability, and pharmacokinetic
testing using mouse models. Over the past four years, our testing center has worked with 10 funded teams to
provide second-site validation of novel genome editor delivery systems, independently evaluating their efficiency
and specificity in reporter models. The Preclinical Mouse Models Core will take full advantage of this exceptional
infrastructure and expertise to achieve the goals of this U19 program. We propose these Specific Aims: 1) To
provide key animal model resources to support the individual disease projects, including optimization of models
and cohort generation; and 2) To execute delivery validation studies that build on proof-of-principle experiments
for each disease project.

## Key facts

- **NIH application ID:** 10842403
- **Project number:** 5U19NS132304-02
- **Recipient organization:** JACKSON LABORATORY
- **Principal Investigator:** Stephen A Murray
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $658,430
- **Award type:** 5
- **Project period:** 2023-05-16 → 2028-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10842403

## Citation

> US National Institutes of Health, RePORTER application 10842403, Preclinical Mouse Model Core (5U19NS132304-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10842403. Licensed CC0.

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