New treatments for Duchenne muscular dystrophy targeting specific gene exons
Official title: Structure-based antisense therapeutics targeting dystrophin exons 44 and 45 for Duchenne Muscular Dystrophy
Funded at University of California Los Angeles · NIH-10871537 · Data updated 2026-11-12
This study is working on new treatments for boys with Duchenne muscular dystrophy (DMD) by creating special medicines that help their bodies make a protein called dystrophin, which is important for muscle function.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $236,250 |
| NIH project number | 1R21HD115071-01 |
| Project ID | NIH-10871537 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are boys diagnosed with Duchenne muscular dystrophy who have mutations affecting exons 44 and 45 of the dystrophin gene.
✕Less likely to help
Patients with Duchenne muscular dystrophy who do not have mutations in exons 44 or 45 may not benefit from this research.
What this research studies
This research focuses on developing innovative antisense oligonucleotide (ASO) therapies to treat Duchenne muscular dystrophy (DMD), a severe genetic disorder affecting muscle function in boys. The approach involves designing ASOs that can skip specific exons in the dystrophin gene, restoring the reading frame and allowing for partial dystrophin expression. By enhancing the binding affinity and specificity of these ASOs through structural interactions, the research aims to improve the effectiveness of existing treatments for a subset of DMD patients.
Why it matters
Potential benefit: If successful, this research could lead to more effective therapies for Duchenne muscular dystrophy, improving muscle function and quality of life for affected patients.
How similar studies have gone: Previous research has shown promise with antisense oligonucleotide therapies for DMD, indicating a potential for success with this novel approach targeting specific exons.
Where this research is happening
-
University of California Los Angeles
Los Angeles, United States Active
Researchers
- Principal investigator: Guo, Feng — University of California Los Angeles
- Study coordinator: Guo, Feng
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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.