Investigating protein balance issues in lymphatic and venous malformations
Protein homeostasis dysfunction in LM and VM pathobiology and therapeutic responses
This study is looking at how problems with proteins in the body can cause issues with blood vessel growth in people with lymphatic and venous malformations, and it aims to find new medications that can help treat these conditions more effectively.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10989889 on NIH RePORTER |
What this research studies
This research focuses on understanding how protein homeostasis dysfunction contributes to lymphatic malformations (LMs) and venous malformations (VMs), which are caused by genetic mutations that activate certain signaling pathways. The team is exploring how these dysfunctions affect the behavior of endothelial cells, which line blood vessels, and how they can lead to severe health issues for patients. By identifying a new class of drugs that inhibit proteasome degradation, the research aims to find more effective treatments that specifically target the underlying problems in these cells. This approach could lead to better therapeutic options for patients suffering from these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients diagnosed with lymphatic malformations or venous malformations, particularly those who have not responded well to existing therapies.
Not a fit: Patients with other types of vascular malformations or those whose conditions are not related to protein homeostasis dysfunction may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of targeted therapies that significantly improve treatment outcomes for patients with lymphatic and venous malformations.
How similar studies have performed: Other research has shown promise in targeting protein homeostasis dysfunction in various diseases, suggesting that this approach could be effective for lymphatic and venous malformations as well.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Shawber, Carrie J. — Columbia University Health Sciences
- Study coordinator: Shawber, Carrie J.
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.