Editing genes to treat rare neurological diseases
Preclinical Genome Editing for Rare Neurological Diseases
['FUNDING_OTHER'] · JACKSON LABORATORY · NIH-11089340
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JACKSON LABORATORY (nih funded) |
| Locations | 1 site (BAR HARBOR, UNITED STATES) |
| Trial ID | NIH-11089340 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Spinal Muscular Atrophy, Friedrich's Ataxia, Huntington's Disease, or Rett Syndrome.
Not a fit: 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.
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 performed: 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
BAR HARBOR, UNITED STATES
- 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 below.
Conditions: Aran-Duchenne disease