How transthyretin proteins clump and harm nerves and the heart
Molecular mechanisms of transthyretin amyloidosis
This project looks at how blood flow and mechanical stress cause transthyretin protein to misfold and form damaging clumps in people with amyloidosis.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scripps Research Institute, the NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11239131 on NIH RePORTER |
What this research studies
Researchers will study transthyretin, a blood protein that can misfold and form amyloid deposits that damage peripheral nerves and the heart. In the lab they will mimic normal body temperature and pH while applying shear forces like those from blood flow to see how wild-type and mutant transthyretin begin to unfold and aggregate. They will use a suite of biophysical tools such as light scattering and other techniques to watch nucleation and fiber growth under physiological conditions. Findings will be related to clinical observations like the link between aortic valve stenosis and cardiac transthyretin amyloidosis to inform future therapies or diagnostics.
Who could benefit from this research
Good fit: Ideal participants are people with diagnosed transthyretin amyloidosis (hereditary or wild-type) or individuals with unexplained cardiomyopathy or aortic valve disease who might donate samples or join related clinical studies.
Not a fit: Patients whose symptoms are caused by non-amyloid conditions or who need immediate clinical treatment are unlikely to gain direct short-term benefit from this basic science work.
Why it matters
Potential benefit: If successful, this work could reveal how to prevent or slow transthyretin aggregation and guide new diagnostics or treatments for ATTR neuropathy and cardiomyopathy.
How similar studies have performed: Prior laboratory studies of transthyretin aggregation have helped develop stabilizing drugs like tafamidis, but directly testing the effect of physiological shear forces on aggregation is a newer approach.
Where this research is happening
La Jolla, United States
- Scripps Research Institute, the — La Jolla, United States (Active)
Researchers
- Principal investigator: Wright, Peter Edwin — Scripps Research Institute, the
- Study coordinator: Wright, Peter Edwin
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.