Editing genes to treat rare neurological diseases
Official title: Preclinical Genome Editing for Rare Neurological Diseases
Funded at Jackson Laboratory · NIH-11089340 · Data updated 2026-11-12
This study is exploring new gene editing methods to find better treatments for rare neurological diseases like Spinal Muscular Atrophy and Huntington's Disease, using advanced tools to carefully change genes in a way that keeps them safe, and testing these methods in mice before thinking about using them in people.
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) | $4,319,346 |
| NIH project number | 5U19NS132304-03 |
| Project ID | NIH-11089340 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Spinal Muscular Atrophy, Friedrich's Ataxia, Huntington's Disease, or Rett Syndrome.
✕Less likely to help
Patients with neurological conditions not included in this research, 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 developing advanced gene editing techniques to create effective treatments for rare neurological diseases such as Spinal Muscular Atrophy, Friedrich's Ataxia, Huntington's Disease, and Rett Syndrome. Using innovative tools like CRISPR/Cas9, the team aims to make precise modifications to the genome without causing significant damage to the DNA. The project includes validating these techniques in mouse models that mimic human conditions, ensuring that the approaches are safe and effective before considering human applications.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking therapies that provide lasting solutions for patients suffering from rare neurological disorders.
How similar studies have gone: Previous research using gene editing technologies has shown promise in treating genetic disorders, indicating a potential for success in this novel approach.
Where this research is happening
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Lutz, Cathleen M — Jackson Laboratory
- Study coordinator: Lutz, Cathleen M
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.