Gene-editing tools in marmosets for brain disorders
Tools for gene editing in marmosets
['FUNDING_U01'] · JOHNS HOPKINS UNIVERSITY · NIH-11127506
This project is creating gene-editing methods in marmosets to build animal models that help researchers understand and develop treatments for human brain disorders.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JOHNS HOPKINS UNIVERSITY (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-11127506 on ClinicalTrials.gov |
What this research studies
This work will develop and refine genome-editing and assisted-reproduction techniques in marmosets, including CRISPR-based editing, embryo culture, and pre-implantation screening. The team will establish marmoset lines carrying tools like Cas9, Cre, and GCaMP for broad or brain‑cell-specific use. Researchers will identify safe genomic sites for inserting these tools and create models with genetic changes relevant to human neurobiology. A dedicated marmoset colony and specialized infrastructure will be used to test behavior and brain function in these models.
Who could benefit from this research
Good fit: This project does not enroll human participants; it focuses on developing animal models and laboratory techniques rather than patient recruitment.
Not a fit: People with brain disorders should not expect direct or immediate clinical benefit because the work is preclinical and centered on animal model development.
Why it matters
Potential benefit: If successful, the tools and marmoset models could speed up research into causes of brain disorders and support development of new treatments.
How similar studies have performed: Gene-editing in non-human primates has shown promise in prior studies but remains technically challenging, so this project builds on and aims to improve those approaches.
Where this research is happening
BALTIMORE, UNITED STATES
- JOHNS HOPKINS UNIVERSITY — BALTIMORE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WANG, XIAOQIN — JOHNS HOPKINS UNIVERSITY
- Study coordinator: WANG, XIAOQIN
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Brain Diseases, Brain Disorders