Therapeutic gene editing for spinal muscular atrophy
Official title: Project 1: Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer)
Funded at Jackson Laboratory · NIH-11326247 · Data updated 2026-11-12
A patient-facing look at a project that aims to test a gene-editing approach to help children with spinal muscular atrophy.
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 | 2026 |
| Award (this year) | $464,183 |
| NIH project number | 5U19NS132304-04 |
| Project ID | NIH-11326247 on NIH RePORTER |
Who could benefit from this research
✓Could help
No specific patient enrollment is described in this grant, as it is a preclinical study using SMA mouse models.
✕Less likely to help
This grant does not involve direct patient participation or treatment at this stage.
What this research studies
This program uses gene editing in mouse models of SMA to optimize treatment methods, study where edits go in the body, and evaluate safety and effectiveness on a larger scale. It collaborates with core facilities to rigorously analyze how the editing works and to prepare data for potential human use. The work is animal-focused but aims to inform future therapies that could benefit people with SMA, including children. If successful, it could pave the way for genome-editing-based therapies for SMA patients in clinical development.
Why it matters
Potential benefit: If successful, the research could advance a gene-editing therapy approach that may one day help children with SMA improve motor function and survival.
How similar studies have gone: Preclinical gene-editing approaches in SMA and other genetic diseases have shown promise in animals and early studies, but clinical translation remains ongoing.
Where this research is happening
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Arbab, Mandana — Jackson Laboratory
- Study coordinator: Arbab, Mandana
Related research
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Correcting Genetic Errors for Spinal Muscular Atrophy
This research looks for new ways to fix the genetic problem that causes Spinal Muscular Atrophy, a condition that weakens muscles.
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Gene editing to treat spinal muscular atrophy
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.
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.