Gene editing to treat Huntington's disease
Official title: Project 3: Therapeutic Gene Editing for Huntington's Disease
Funded at Jackson Laboratory · NIH-11089357 · Data updated 2026-11-12
This study is exploring new ways to use gene editing to help people with Huntington's disease by fixing the genetic mistakes that cause it, with the hope of slowing down or stopping the disease's effects.
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) | $666,838 |
| NIH project number | 5U19NS132304-03 |
| Project ID | NIH-11089357 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Huntington's disease, particularly those in the early stages of the condition.
✕Less likely to help
Patients with advanced Huntington's disease or those who do not have the specific genetic mutation targeted by this research may not benefit.
What this research studies
This research focuses on developing advanced gene editing techniques to address Huntington's disease, a genetic disorder that leads to severe neurological decline. The project aims to optimize methods for correcting the genetic mutations responsible for the disease using innovative technologies like base editing and prime editing. By utilizing adeno-associated viral vectors for delivery, the research seeks to enhance the effectiveness of these gene editing strategies in pre-clinical models. Ultimately, the goal is to create a potential therapeutic approach that could slow or halt disease progression in affected individuals.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that may significantly improve the quality of life and longevity for patients with Huntington's disease.
How similar studies have gone: Previous research in gene editing has shown promising results in other genetic disorders, suggesting potential success for similar approaches in Huntington's disease.
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
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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.