How myostatin and related proteins control muscle growth
Signaling mechanisms utilized by myostatin and related ligands in skeletal muscle
Researchers are learning how myostatin and similar proteins control muscle size to help people with muscle-wasting conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Connecticut Sch of Med/dnt NIH-funded |
| Lab location | 1 site (Farmington, United States) |
| Project ID | NIH-11258907 on NIH RePORTER |
What this research studies
This lab uses genetically modified mice to turn off or alter specific parts of the myostatin signaling pathway in skeletal muscle and then measures how those changes affect muscle size and function. The team focuses on the receptors (ACVR2/ACVR2B and ALK4/ALK5) and the downstream SMAD2/SMAD3 proteins, but also looks for other signaling routes that might explain unexpected results. By combining biochemical studies with targeted gene deletions in muscle fibers, they aim to map which components are most important for limiting muscle growth. The work is meant to reveal new targets that could eventually lead to therapies for people who lose muscle.
Who could benefit from this research
Good fit: Although this project does not enroll people, its findings are most relevant to individuals with muscle-wasting conditions such as muscular dystrophy, sarcopenia, or cachexia who might benefit from future treatments targeting the myostatin pathway.
Not a fit: People with health issues unrelated to muscle loss or whose muscle problems arise from causes not involving myostatin signaling are less likely to benefit from this specific line of work.
Why it matters
Potential benefit: If successful, this work could point to new ways to increase or preserve muscle mass in conditions like muscular dystrophy, age-related frailty, or cancer-related wasting.
How similar studies have performed: Animal studies blocking myostatin or its receptors have increased muscle mass, while early human trials showed mixed results with some gains in size but limited functional benefit and safety questions, so this research aims to clarify the biology behind those outcomes.
Where this research is happening
Farmington, United States
- University of Connecticut Sch of Med/dnt — Farmington, United States (Active)
Researchers
- Principal investigator: Lee, Se-Jin — University of Connecticut Sch of Med/dnt
- Study coordinator: Lee, Se-Jin
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 below.