Progranulin and lysosome function in frontotemporal brain disease
Lysosomal Function of Progranulin and Neurodegeneration
This research looks at how pieces of the progranulin protein help lysosomes work in the brain to better understand frontotemporal lobar degeneration.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cornell University NIH-funded |
| Lab location | 1 site (Ithaca, United States) |
| Project ID | NIH-11320756 on NIH RePORTER |
What this research studies
From a patient's point of view, scientists are using new antibodies to track individual progranulin fragments inside lysosomes and see how they interact with other lysosomal proteins. The team studies these interactions using lab models and biochemical tests and compares findings to changes seen when progranulin is missing. They have found specific links between one granulin fragment and the lysosomal protein CD68, and between progranulin and the enzyme NAGA, and are mapping these connections. The goal is to learn which fragments and partners are most important for lysosome health in the brain.
Who could benefit from this research
Good fit: People with frontotemporal lobar degeneration, especially those who carry mutations in the GRN gene, would be the most directly relevant candidates for related clinical work.
Not a fit: People whose cognitive problems come from unrelated causes (for example Alzheimer's disease without progranulin involvement) are unlikely to see direct benefit from this specific line of research.
Why it matters
Potential benefit: If successful, this work could point to specific progranulin fragments or lysosomal proteins to target with therapies that slow or prevent frontotemporal dementia.
How similar studies have performed: Previous research linked progranulin loss to lysosomal dysfunction and frontotemporal dementia, but the idea that individual granulin peptides have unique lysosomal roles is a newer and less-tested area.
Where this research is happening
Ithaca, United States
- Cornell University — Ithaca, United States (Active)
Researchers
- Principal investigator: Hu, Fenghua — Cornell University
- Study coordinator: Hu, Fenghua
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.