# Establishment of Somatic Cell Nuclear Transfer as a Universal Platform for Cloning Marmosets

> **NIH NIH R21** · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · 2022 · $220,800

## Abstract

PROJECT SUMMARY/ABSTRACT
The common marmoset (Callithrix jacchus) is a New World non-human primate (NHP) with several practical
advantages in biomedical research. Due to their phylogenetic proximity, marmosets are a genetically diverse
NHP species with similar physiological, metabolic, and immunological functions as humans. Marmosets retain
the typical anatomical and functional organization of the human brain. Marmosets have complex cognitive and
social behavior. The above characteristics place marmosets as an ideal NHP model to bridge the gap between
mice and humans for both basic and translational neuroscience. Marmosets reach sexual maturity at circa 18
months and give birth to multiple infants twice a year. Their short gestation period and compatibility with gene
editing techniques make marmosets ideally poised to become the NHP model of choice in studying the genetic
causes of neurological and neuropsychiatric disorders and understanding brain function. We have successfully
generated transgenic marmosets expressing genetically encoded calcium indicators and genetically engineered
marmosets with NOTCH3 mutations that cause the small vessel disease CADASIL. We have also made
marmosets harboring PSEN1 mutations that cause early-onset Alzheimer’s disease. Our gene-edited
marmosets will enable us to investigate the genetic causes of chronic neurological disorders. However, two main
issues hinder the broader availability of genetically engineered marmosets. First, lentiviral-based approaches
suffer from an uncontrollable integration of transgene with variation in copy number, and nuclease-based gene
editing produces embryos with mosaic editing, leading to unpredictable gene expression patterns and variable
phenotype. Second, and most importantly, there are no efficient ways to propagate an individual showing the
interest phenotype. This proposal addresses these shortcomings by developing and optimizing somatic cell
nuclear transfer (SCNT) as a universal platform for cloning marmosets. We want to build on our vast experience
generating genetically modified marmosets to (1) develop and optimize the enucleation procedure in marmoset
somatic cell nuclear transfer and (2) develop and optimize approaches enabling technologies for efficient
epigenetic reprogramming during somatic cell nuclear transfer in the marmoset. These results will establish
optimized marmoset SCNT protocols and unravel a novel enabling universal platform to generate a sizeable
number of cloned marmosets expressing any phenotype of interest. This platform will significantly facilitate the
propagation and sharing of marmoset models for neuroscience and translational research supported by multiple
NIH ICs.

## Key facts

- **NIH application ID:** 10510648
- **Project number:** 1R21OD033666-01
- **Recipient organization:** UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- **Principal Investigator:** Jung Eun Park
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $220,800
- **Award type:** 1
- **Project period:** 2022-08-15 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10510648

## Citation

> US National Institutes of Health, RePORTER application 10510648, Establishment of Somatic Cell Nuclear Transfer as a Universal Platform for Cloning Marmosets (1R21OD033666-01). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10510648. Licensed CC0.

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