Investigating the effects of antisense oligonucleotides on spinal muscular atrophy treatment
Official title: Transcriptome-Wide Effect of Therapeutic Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy
Funded at Iowa State University · NIH-11078218 · Data updated 2026-11-12
This study is looking at a new way to help people with spinal muscular atrophy (SMA) by testing special treatments that could improve how a key gene works, with the hope of finding better therapies to make life easier for SMA patients.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Iowa State University NIH-funded |
| Lab location | 1 site (Ames, United States) |
| Project start | May 1, 2024 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $76,500 |
| NIH project number | 5R03NS136717-02 |
| Project ID | NIH-11078218 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include infants and children diagnosed with spinal muscular atrophy, particularly those who have not responded adequately to current therapies.
✕Less likely to help
Patients with forms of spinal muscular atrophy that are not amenable to treatment with antisense oligonucleotides may not benefit from this research.
What this research studies
This research focuses on spinal muscular atrophy (SMA), a genetic condition that leads to severe muscle weakness and is a leading cause of infant mortality. The study aims to explore the use of antisense oligonucleotides (ASOs) to prevent the skipping of a crucial part of the SMN2 gene, which is essential for producing the SMN protein. By understanding how different chemical modifications of ASOs affect their performance, the research seeks to improve existing therapies and potentially develop new treatment options for SMA patients. Patients may benefit from insights into more effective therapies that could enhance their quality of life.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for spinal muscular atrophy, improving patient outcomes and quality of life.
How similar studies have gone: Previous research has shown success with antisense oligonucleotide therapies for spinal muscular atrophy, indicating a promising avenue for further exploration.
Where this research is happening
-
Iowa State University
Ames, United States Active
Researchers
- Principal investigator: Singh, Ravindra N — Iowa State University
- Study coordinator: Singh, Ravindra N
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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.