How transthyretin proteins clump and harm nerves and the heart
Official title: Molecular mechanisms of transthyretin amyloidosis
Funded at Scripps Research Institute, the · NIH-11239131 · Data updated 2026-11-12
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 start | April 1, 2020 |
| Project end | December 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $566,635 |
| NIH project number | 5R01DK124211-06 |
| Project ID | NIH-11239131 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
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.
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.
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 gone: 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
-
Scripps Research Institute, the
La Jolla, United States Active
Researchers
- Principal investigator: Wright, Peter Edwin — Scripps Research Institute, the
- Study coordinator: Wright, Peter Edwin
Related research
Other NIH-funded projects on the same topics.
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Understanding how kidney-like blood flow may cause amyloid buildup
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Blood test to detect ATTR amyloidosis earlier using a smart detection kit
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Detection and measurement of amyloid in people carrying the V122I TTR gene variant
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Identifying Hidden Cardiac Amyloidosis in Black V122I TTR Carriers
This project looks for early signs of heart damage in Black individuals who carry the V122I TTR variant and may benefit from new treatments if caught early.
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Chip phosphorylation boosts breakdown of mutant TTR to help heart amyloidosis
A scientist-led project aims to help people with a heart disease caused by a mutant protein called transthyretin (TTR) by boosting the body’s protein-cleanup systems to remove the harmful TTR deposits.
-
Finding transthyretin heart amyloid from lumbar spine surgery tissue
This project looks for transthyretin (TTR) amyloid in tissue removed during lumbar spine surgery to find people who may later develop TTR cardiac amyloidosis.
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.