# Enhancing The Value of Pigs for Agriculture and Biomedical Applications By Using Novel Genome Editing Strategies

> **NIH NIH R01** · UNIVERSITY OF MISSOURI-COLUMBIA · 2024 · $766,737

## Abstract

Project Summary/Abstract
Genetically engineered (GE) animal models are essential for generating biomedical models for human disease
and for gaining a better understanding of animal biology. Targeted modification of the animal genome allows the
animals to present human disease phenotypes, and therefore, are critical to design and develop novel treatments.
The use of GE large animal models such as pigs often results in clinically relevant outcomes as their physiology
and anatomy resemble humans. For example, introducing genetic elements responsible for cystic fibrosis and
immunodeficiency to the pig genome induces GE pigs to closely recapitulate symptoms of the diseases. However,
GE pigs are not widely available in biomedicine due to the amount of time required to establish such models. As
a large animal species, a single round of breeding in pigs takes at least one year and often multiple rounds of
breeding is necessary to establish GE pig models. Application of genome editing tools, such as the CRISPR/Cas
system, has significantly improved efficacy to introduce targeted modifications to the pig genome. However,
concerns over unintended genome alterations from genome editing procedure and days required to introduce
targeted modifications in pigs as a large animal model impedes wide use of the technology. Our objective of
this project is to evaluate the efficacy and safety of genome editing technology and design novel approaches
that will assist in rapid phenotyping of animal models after a targeted genome editing event. Three specific aims
are proposed to reach our goal. First, we will generate methods for global detection of off-targeting events in GE
pigs. Secondly, we will develop strategies to secure genome integrity during the genome editing process. Finally,
we propose to develop a strategy to rapidly phenotype GE fetuses and to modify the genome of wild-type pigs.
Targets of this third aim are genes associated with traits that are relevant in both agriculture and biomedicine.
The knowledge obtained from this project can be implemented to expand the use of GE pigs in biomedicine while
also having an impact on agriculture production. The importance of using the genome to predict the phenotype
for rapid identification of improved alleles and traits will be grown here. Our expertise in using genome editing
technology and GE pig models will be employed to complete the proposed aims. Outcomes of this project should
increase the availability of GE pig models in biomedicine and agriculture by effectively capturing subsequent
phenotypes after genome editing events. We propose to utilize pigs as a model to investigate the efficacy of the
proposed strategies; however, our findings should be easily transferred to producing other animal models in
biomedicine and agriculturally important species, as well. Given the importance of pigs used as animal models,
our findings should be beneficial to both NIH and USDA agencies.

## Key facts

- **NIH application ID:** 10868598
- **Project number:** 5R01OD035561-02
- **Recipient organization:** UNIVERSITY OF MISSOURI-COLUMBIA
- **Principal Investigator:** Kiho Lee
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $766,737
- **Award type:** 5
- **Project period:** 2023-07-01 → 2027-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10868598

## Citation

> US National Institutes of Health, RePORTER application 10868598, Enhancing The Value of Pigs for Agriculture and Biomedical Applications By Using Novel Genome Editing Strategies (5R01OD035561-02). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10868598. Licensed CC0.

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