How transthyretin (TTR) harms heart and nerve tissue in amyloid disease
Probing the biochemical mechanism of amyloid disease
This work looks at how abnormal transthyretin proteins form toxic clumps that damage heart and nerve tissue in people with hereditary or age-related TTR 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-11168995 on NIH RePORTER |
What this research studies
From a patient point of view, researchers are examining the exact shapes and locations of TTR protein aggregates in human heart tissue using advanced 3D tissue-clearing and imaging methods. They combine that 3D pathology with biochemical studies to find which aggregate types cause cell dysfunction and death. The team aims to link those toxic structures to early biomarkers that could show whether a therapy is working. Ultimately the project is intended to point to new therapeutic targets and improve how we detect and track TTR amyloid disease.
Who could benefit from this research
Good fit: People with hereditary (mutant) or wild-type transthyretin cardiomyopathy or TTR-related neuropathy, especially those willing to provide clinical samples or participate in tissue-based studies, would be most relevant.
Not a fit: Patients with non-TTR forms of amyloidosis (for example AL amyloidosis) or people needing immediate clinical therapy rather than contributing samples are unlikely to gain direct benefit from this project.
Why it matters
Potential benefit: If successful, this work could reveal which TTR aggregates cause damage and enable earlier tests and better-targeted treatments for TTR amyloidosis.
How similar studies have performed: Drugs that stabilize TTR, such as tafamidis, have improved outcomes for some patients, but the precise link between specific aggregate structures and tissue damage remains unclear, so this detailed structural approach is relatively novel.
Where this research is happening
La Jolla, United States
- Scripps Research Institute, the — La Jolla, United States (Active)
Researchers
- Principal investigator: Kelly, Jeffery W — Scripps Research Institute, the
- Study coordinator: Kelly, Jeffery W
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.