Developing advanced gene editing techniques for therapeutic use
Official title: Gene Editing Core
Funded at Jackson Laboratory · NIH-11089346 · Data updated 2026-11-12
This study is working on improving gene editing techniques to make treatments for genetic conditions safer and more effective, which could help patients get better care.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Jackson Laboratory NIH-funded |
| Lab location | 1 site (Bar Harbor, United States) |
| Project start | May 16, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $403,425 |
| NIH project number | 5U19NS132304-03 |
| Project ID | NIH-11089346 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic conditions that may be addressed through gene editing technologies.
✕Less likely to help
Patients with conditions that are not amenable to gene editing or those who do not have a genetic basis for their condition may not benefit from this research.
What this research studies
This research focuses on creating and optimizing gene editing strategies to improve therapeutic outcomes while reducing unwanted effects. The Gene Editing Core at the Broad Institute of MIT and Harvard will assess the effectiveness of these techniques on isolated cell lines and animal tissues. By analyzing both on-target and off-target editing activities, the project aims to refine gene editing technologies for specific therapeutic targets. Patients may benefit from the advancements in gene editing that could lead to more effective treatments for various genetic conditions.
Why it matters
Potential benefit: If successful, this research could lead to more precise and effective gene therapies for patients with genetic disorders.
How similar studies have gone: Previous research in gene editing has shown promising results, indicating that this approach has the potential for significant advancements in treatment.
Where this research is happening
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Liu, David R — Jackson Laboratory
- Study coordinator: Liu, David R
Related research
Other NIH-funded projects on the same topics.
-
Mouse Phenome Project: a shared mouse data resource for understanding human disease
A large, centralized database of mouse phenotypes is created to help researchers study human diseases by comparing mouse and human biology.
-
Pediatric Cancer Drug Testing Coordinating Center
A coordinating center is coordinating how young patients’ cancer models are tested with targeted drugs to help improve pediatric cancer outcomes.
-
Increasing the usefulness of pediatric genomic medicine with Exomiser
A pediatric genetic diagnosis project is improving how we use sequencing data to find rare disease causes in children.
-
Modeling Hearing and Balance Disorders in Mice: HEar@JAX Workshop
A five-day workshop at The Jackson Laboratory trains researchers in using mouse models to study hearing and balance, with hands-on labs for 20 participants.
-
Age-stratified gut-on-a-chip model to study microbiome-driven aging of the lining of the intestine
A human gut-on-a-chip model will be created to study how aging-related changes in the gut lining interact with microbiome shifts, using patient-derived tissues and age-appropriate microbes.
-
Single-cell long-read sequencing to map cancer RNA variants
A cancer research grant aims to make advanced single-cell RNA sequencing more accurate and widely usable to understand how different RNA isoforms affect brain and breast cancers.
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.