# Rhesus Macaque Somatic Cell Gene Editing Resource

> **NIH NIH U24** · OREGON HEALTH & SCIENCE UNIVERSITY · 2020 · $811,732

## Abstract

SUMMARY
Until recently, the ability to create specific genetic alterations in mammals was primarily restricted to using
homologous recombination in mice. Over the past decade, new developments in genome editing opened the
door for introducing targeted changes to the DNA of most species, including humans. New editing techniques
rely on the error-prone repair processes nonhomologous-end joining (NHEJ) and microhomology mediated-
end joining (MMEJ) to generate small insertions or deletions within a target gene after the creation of a double
stranded DNA break, which in turn may lead to a premature stop codon or a nonfunctional protein.
Alternatively, a desired specific DNA sequence can be integrated at the site of the DNA lesion through
homology-directed repair (HDR). Other systems also have been developed that allow for the modification of a
single nucleotide through DNA base editing (DBE), thereby avoiding the creation of double stranded DNA
breaks. While these approaches are major advances toward correcting disease-causing inherited mutations in
embryos or de novo mutations that occur in a tissue specific manner, there are concerns regarding efficacy
and safety. Genome editing methods have the potential to create a “mosaic” of different mutations, some that
may be corrective and some that may be detrimental to cellular function. Another concern includes editing in
homologous “off-target” DNA sequences, which in turn can have negative impacts on the function of an
unrelated gene or set of genes. Therefore, it is imperative that a reporter animal is created that can be used to
test editing heterogeneity and off-target effects for existing or yet to be developed genome editing techniques
that would potentially be used for therapeutic applications. To accomplish this objective, we propose to create
a rhesus macaque reporter animal through two complimentary approaches that will provide investigators with
the resources needed for assessing the efficiency and specificity of NHEJ-, MMEJ-, HDR-, or DBE-based
approaches. We will generate personalized genome assemblies from each reporter animal to facilitate highly
accurate detection of subsequent off-target effects. A cohort of animals will be derived that will serve as the
foundation for subsequent reporter assessment projects. Importantly, based on the close genetic,
physiological, and anatomical relationship with humans, assessment of genome editing activities in these
rhesus macaque reporter animals will provide insight into their therapeutic potential.

## Key facts

- **NIH application ID:** 9978950
- **Project number:** 5U24OD026631-03
- **Recipient organization:** OREGON HEALTH & SCIENCE UNIVERSITY
- **Principal Investigator:** Jon D Hennebold
- **Activity code:** U24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $811,732
- **Award type:** 5
- **Project period:** 2018-09-19 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9978950

## Citation

> US National Institutes of Health, RePORTER application 9978950, Rhesus Macaque Somatic Cell Gene Editing Resource (5U24OD026631-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9978950. Licensed CC0.

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