# IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE

> **NIH NIH U19** · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $315,720

## Abstract

PROJECT SUMMARY
Many monogenic diseases strike prior to or soon after birth and cause significant morbidity and mortality. In utero
genome editing (IUGE) takes advantage of normal fetal properties, including small size, abundant progenitor
cells, and immature immune system, to safely and efficiently institute the genome editing therapeutic prior to the
onset of pathology. To fully understand the potential of IUGE and in preparation for IND applications, rigorous
small and large animal studies are required to assess efficacy as well as fetal and maternal safety. The In
Utero Small and Large Animal Resource Core will provide the knowledge and technical expertise required to
design, implement and analyze in vivo gene editing therapies in mouse and large animal fetal models, including
nonhuman primates.

## Key facts

- **NIH application ID:** 10896259
- **Project number:** 5U19NS132301-02
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** Alan W. Flake
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $315,720
- **Award type:** 5
- **Project period:** 2023-08-01 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10896259

## Citation

> US National Institutes of Health, RePORTER application 10896259, IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE (5U19NS132301-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10896259. Licensed CC0.

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