Structure-guided antisense treatment for Duchenne with exon 44 or 45 mutations
Official title: Structure-based antisense therapeutics targeting dystrophin exons 44 and 45 for Duchenne Muscular Dystrophy
Funded at University of California Los Angeles · NIH-11174566 · Data updated 2026-11-12
Specially designed antisense molecules aim to boost dystrophin production in people with Duchenne whose genetic mutations can be fixed by skipping exon 44 or 45.
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 | 2025 |
| Award (this year) | $196,875 |
| NIH project number | 5R21HD115071-02 |
| Project ID | NIH-11174566 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with genetically confirmed Duchenne muscular dystrophy whose mutations are correctable by skipping exon 44 or exon 45 (together about 14% of DMD cases) would be the ideal candidates.
✕Less likely to help
Patients whose mutations are not in exon 44 or 45, or those with very advanced, irreversible muscle loss, are unlikely to benefit from these specific ASOs.
What this research studies
Researchers are designing antisense oligonucleotides (ASOs) that match not just the RNA sequence but its 3‑D shape so the drug can bind more tightly and precisely to dystrophin pre-mRNA. The project focuses on creating ASOs that encourage cells to skip exon 44 or 45 so the dystrophin protein can be made in a working form. Scientists will use structural design, chemistry changes, and laboratory testing (including crystal structures for the ASO–exon complex) to guide which molecules look most promising. The goal is to create ASOs with higher activity and better delivery than current drugs for the ~14% of DMD patients with these exon targets.
Why it matters
Potential benefit: If successful, this approach could raise dystrophin levels and improve muscle function for patients with exon 44 or 45–amenable Duchenne mutations.
How similar studies have gone: A few ASO drugs for DMD have FDA approval but have shown limited clinical benefit, and this structure‑based design represents a new, less‑tested approach to improve efficacy.
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.