Gene editing to treat spinal muscular atrophy
Project 1: Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer)
This study is working on a new gene editing treatment for spinal muscular atrophy (SMA) to see how well it can help improve muscle strength and overall health, especially for infants affected by this condition.
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-11089352 on NIH RePORTER |
What this research studies
This research focuses on developing a gene editing therapy for spinal muscular atrophy (SMA), a severe genetic disorder that leads to muscle weakness and is a leading cause of infant mortality. The project aims to optimize treatment protocols using advanced gene editing techniques in mouse models of SMA, assess the safety and effectiveness of these treatments, and conduct large-scale studies to evaluate their potential. By closely analyzing the outcomes of these gene editing approaches, the research seeks to pave the way for future clinical applications that could significantly improve patient outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are infants diagnosed with spinal muscular atrophy, particularly those with the most common type, SMA type I.
Not a fit: Patients with other forms of motor neuron diseases or those without a diagnosis of spinal muscular atrophy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that significantly improves the survival and quality of life for infants with spinal muscular atrophy.
How similar studies have performed: Previous research using gene editing techniques has shown promise in treating genetic disorders, indicating that this approach could be effective for spinal muscular atrophy as well.
Where this research is happening
Bar Harbor, United States
- Jackson Laboratory — Bar Harbor, United States (Active)
Researchers
- Principal investigator: Arbab, Mandana — Jackson Laboratory
- Study coordinator: Arbab, Mandana
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.