# Tools for gene editing in marmosets

> **NIH NIH U01** · JOHNS HOPKINS UNIVERSITY · 2024 · $1,955,019

## Abstract

Tools for Gene Editing in Marmosets
SUMMARY
 The goal of this proposal is to create an efficient new set of tools and techniques for the generation of
genetically modified marmosets suitable for use as models of brain physiology and function. Development of
efficient methods of genetic manipulation in the species will require refinement of genome editing technologies,
breeding technologies, ovarian stimulation and oocyte retrieval, embryo culture, and pre-implantation screening
of embryos for the desired genetic alternations. A large colony of marmosets will be necessary, along with an
infrastructure to refine technologies and an interdisciplinary team with expertise in marmoset care, assisted
reproduction technologies, tissue-specific expression with Cre-drivers and CRISPR technologies, and behavior
testing and neuropsychiatric analysis. The Specific Aims of this proposal are to: (1) Improve technology for in
vitro genetic manipulation of marmosets and explore novel methods for marmoset gene editing; (2) Identify safe
harbor loci and establish Cas9, Cre, and GCaMP marmoset lines using promoters for ubiquitous expression,
selective expression in brain, or regional or cell-type specific expression; and (3) Generate marmoset models
with genetic alterations relevant to the neurobiology of human brain diseases. These studies will provide tools
for future gene editing experiments in marmosets, will establish an initial set of genetically modified tool lines for
use in the field, and will provide initial proof-of-principle lines for marmoset models of genetic alterations
potentially relevant to the neurobiology of human brain diseases.

## Key facts

- **NIH application ID:** 10892191
- **Project number:** 5U01DA056556-03
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Richard L Huganir
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $1,955,019
- **Award type:** 5
- **Project period:** 2022-09-01 → 2027-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10892191

## Citation

> US National Institutes of Health, RePORTER application 10892191, Tools for gene editing in marmosets (5U01DA056556-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10892191. Licensed CC0.

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