Gene editing to treat spinal muscular atrophy
Official title: Project 1: Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer)
Funded at Jackson Laboratory · NIH-11089352 · Data updated 2026-11-12
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 start | May 16, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $548,113 |
| NIH project number | 5U19NS132304-03 |
| Project ID | NIH-11089352 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants diagnosed with spinal muscular atrophy, particularly those with the most common type, SMA type I.
✕Less likely to help
Patients with other forms of motor neuron diseases or those without a diagnosis of spinal muscular atrophy may not benefit from this research.
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.
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 gone: 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
-
Jackson Laboratory
Bar Harbor, United States Active
Researchers
- Principal investigator: Arbab, Mandana — Jackson Laboratory
- Study coordinator: Arbab, Mandana
Related research
Other NIH-funded projects on the same topics.
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Genetic clues to mouth muscles development in SMA
A small NIH grant studies how nerve cells controlling jaw movements develop normally and how this process goes wrong in spinal muscular atrophy (SMA).
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Therapeutic gene editing for spinal muscular atrophy
A patient-facing look at a project that aims to test a gene-editing approach to help children with spinal muscular atrophy.
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SMN-independent approaches for spinal muscular atrophy
This project looks for new treatments for people with spinal muscular atrophy that work without relying only on restoring the SMN protein.
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How RNA problems cause nerve and muscle damage in spinal muscular atrophy
This project looks at how RNA-related problems lead to nerve and muscle damage in people with spinal muscular atrophy to help find better treatment targets.
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Investigating speech development in children with spinal muscular atrophy after new therapies
This study is looking at how new treatments for spinal muscular atrophy (SMA) can help improve speech and communication skills in children and young adults aged 2 to 21, by comparing their speech abilities to what’s expected for their age.
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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.
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.