Gene editing to treat Huntington's disease
Project 3: Therapeutic Gene Editing for Huntington's Disease
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 ID | NIH-11089357 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Huntington's disease, particularly those in the early stages of the condition.
Not a fit: Patients with advanced Huntington's disease or those who do not have the specific genetic mutation targeted by this research may not benefit.
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 performed: 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
Bar Harbor, United States
- Jackson Laboratory — Bar Harbor, United States (Active)
Researchers
- Principal investigator: Liu, David R — Jackson Laboratory
- Study coordinator: Liu, David R
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.