Developing new treatments for Duchenne Muscular Dystrophy using oligonucleotides
Official title: Development of Utrophin Site Blocking Oligos (SBOs) to Treat Duchenne Muscular Dystrophy (DMD)
Funded at University of Pennsylvania · NIH-10678195 · Data updated 2026-11-12
This study is looking for a new way to help boys with Duchenne Muscular Dystrophy by using special treatments that could boost a protein called utrophin to improve their muscle strength and health.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | August 18, 2023 |
| Project end | July 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $490,761 |
| NIH project number | 1UG3NS132420-01 |
| Project ID | NIH-10678195 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are boys diagnosed with Duchenne Muscular Dystrophy, particularly those who are in the early stages of the disease.
✕Less likely to help
Patients with other forms of muscular dystrophy or those who are in advanced stages of Duchenne Muscular Dystrophy may not benefit from this research.
What this research studies
This research focuses on finding a new treatment for Duchenne Muscular Dystrophy (DMD), a severe genetic disorder that primarily affects boys. The approach involves using specially designed oligonucleotides to block certain microRNAs that inhibit the production of utrophin, a protein that can compensate for the missing dystrophin in DMD patients. By increasing utrophin levels, the research aims to improve muscle function and overall health in individuals with DMD. The study will involve testing these oligonucleotides in animal models to assess their effectiveness and safety before considering human trials.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that improves muscle function and quality of life for patients with Duchenne Muscular Dystrophy.
How similar studies have gone: Previous research has shown promise in using similar oligonucleotide approaches to increase utrophin levels, indicating a potential for success in this novel treatment strategy.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Khurana, Tejvir S — University of Pennsylvania
- Study coordinator: Khurana, Tejvir S
Related research
Other NIH-funded projects on the same topics.
-
3D printed human heart model for Duchenne muscular dystrophy (DMD) cardiomyopathy progression
A patient-friendly 3D-printed heart model using stem cells and gene editing helps study how DMD affects the heart over time and tests potential therapies.
-
Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
-
Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
-
Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
-
MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
-
Digital motor-skill tracking for infants with Duchenne Muscular Dystrophy using wearables
A wearable-sensor project aims to quietly track and quantify how motor skills develop in babies and young boys with Duchenne Muscular Dystrophy.
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.