Protecting brain cells in protein-clumping diseases by boosting mitochondria and natural protein helpers
Official title: Slowing proteotoxic neurodegeneration by boosting mitochondrial bioenergetics and recruiting a novel class of chaperones
Funded at Miami VA Health Care System · NIH-11212791 · Data updated 2026-11-12
This project aims to protect brain cells in people with protein-misfolding diseases like Huntington's and Parkinson's by improving mitochondrial energy and recruiting natural protein 'chaperone' enzymes.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Miami VA Health Care System NIH-funded |
| Lab location | 1 site (Miami, United States) |
| Project start | January 1, 2017 |
| Project end | September 30, 2026 |
| Funding year | 2026 |
| NIH project number | 5I01BX003303-08 |
| Project ID | NIH-11212791 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with Huntington's disease, Parkinson's disease, or other neurodegenerative disorders caused by toxic protein aggregation would be the most relevant candidates for future therapies arising from this work.
✕Less likely to help
Patients with conditions that are not driven by protein misfolding or those with very advanced neuronal loss may be unlikely to benefit directly from these approaches.
What this research studies
Researchers are studying how boosting the cell's energy factories (mitochondria) and turning on everyday enzymes to act as protein "helpers" can prevent harmful protein clumps in neurons. They use yeast, fruit fly, and mouse models to find which enzymes in the NAD+ salvage pathway can act as chaperones and protect cells under proteotoxic stress. The team tests whether increasing mitochondrial biogenesis or overexpressing enzymes such as NMNAT/NMA1, NADS/Qns1, NaPTRase/Npt1 and NDase/Pnc1 reduces protein damage and improves neuron survival. Results could point to new treatment strategies that restore energy balance and protein folding in neurodegenerative disorders.
Why it matters
Potential benefit: If successful, this work could lead to therapies that reduce toxic protein buildup and slow cognitive and movement decline in disorders like Huntington's and Parkinson's.
How similar studies have gone: Related work in yeast, fly and some mouse models has shown that boosting mitochondrial function and NMNAT-family enzymes can protect against proteotoxicity, but translation to humans remains unproven.
Where this research is happening
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Miami VA Health Care System
Miami, United States Active
Researchers
- Principal investigator: Barrientos, Antoni — Miami VA Health Care System
- Study coordinator: Barrientos, Antoni
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.