Antisense therapy for MC4R-related genetic obesity in young children
Official title: Antisense Oligonucleotide-Based Therapeutic Strategy for Genetic Obesity Caused by MC4R Mutations
Funded at Saint Louis University · NIH-11380445 · Data updated 2026-11-12
A new antisense oligonucleotide treatment is being explored to help children with MC4R-related obesity, aiming to reduce excessive hunger and improve weight-related health risks.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Saint Louis University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project start | June 9, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $423,500 |
| NIH project number | 1R21HD117251-01A1 |
| Project ID | NIH-11380445 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children under 10 years old with MC4R-related obesity.
✕Less likely to help
Children without MC4R-related obesity or those outside the target age range may not benefit.
What this research studies
This grant focuses on a genetic form of obesity caused by MC4R mutations and how a targeted antisense therapy might modulate the pathway that controls appetite. Researchers will examine how to design a safe ASO approach to correct or mitigate the genetic driver in affected individuals. The study emphasizes a translational path from molecular concepts toward a potential pediatric treatment, with attention to safety, dosing, and early efficacy signals. If successful, the work could pave the way for a new option for children under 10 who currently lack approved therapies. The project spans from molecular design to preclinical and early human-relevant studies to inform future trials.
Why it matters
Potential benefit: If successful, the therapy could reduce hunger and obesity-related health risks in children with MC4R mutations.
How similar studies have gone: Other MC4R-focused approaches have progressed, but this A SO strategy in young children is novel and exploratory.
Where this research is happening
-
Saint Louis University
Saint Louis, United States Active
Researchers
- Principal investigator: Nguyen, Andrew D. — Saint Louis University
- Study coordinator: Nguyen, Andrew D.
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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.