New treatment approaches for Loeys-Dietz aortic aneurysms using human cell–based models
Therapeutic Insights into Loeys-Dietz Syndrome using Humanized Models
This project works to create and test medicines aimed at preventing or slowing aortic root aneurysms in people with Loeys-Dietz Syndrome.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project ID | NIH-11311070 on NIH RePORTER |
What this research studies
If you take part, researchers use cells taken from people to make patient-derived stem cells and edit disease-causing genes with CRISPR to recreate the Loeys-Dietz blood vessel problem. Those human cells are formed into bioengineered vascular grafts and studied in controlled lab and animal settings to see how the aortic root weakens and which treatments stop dilation. The team will screen candidate drugs and study how they affect collagen enzymes and mitochondrial health tied to aneurysm growth. Findings are intended to identify therapies that could move toward testing in people.
Who could benefit from this research
Good fit: Ideal candidates are people with genetically confirmed Loeys-Dietz Syndrome or a strong clinical diagnosis who might consider donating cells or samples for research.
Not a fit: People without Loeys-Dietz Syndrome or whose aneurysms are caused by unrelated conditions are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this work could lead to medicines that slow or prevent aortic root aneurysms in Loeys-Dietz patients and reduce the need for early surgery.
How similar studies have performed: Prior human cell-based models and animal graft work have clarified disease mechanisms, but using these humanized models specifically to screen therapies for Loeys-Dietz is a relatively new approach.
Where this research is happening
Ann Arbor, United States
- University of Michigan at Ann Arbor — Ann Arbor, United States (Active)
Researchers
- Principal investigator: Yang, Bo — University of Michigan at Ann Arbor
- Study coordinator: Yang, Bo
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.