A gene therapy tool to clear toxic proteins in neurodegenerative diseases
Official title: Autophagon: an Autophagy-Functionalizing Gene Therapy Tool for Neurodegenerative Diseases
Funded at Brigham and Women's Hospital · NIH-11001449 · Data updated 2026-11-12
This study is testing a new gene therapy called Autophagon that aims to help people with Alzheimer's and Parkinson's by clearing out harmful proteins in the brain, using a special delivery method to help the body remove these toxins naturally.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | March 15, 2022 |
| Project end | December 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $688,634 |
| NIH project number | 5R01AG074899-05 |
| Project ID | NIH-11001449 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Alzheimer's or Parkinson's disease, particularly those with genetic mutations that contribute to protein aggregation.
✕Less likely to help
Patients with neurodegenerative diseases not associated with Amyloid-β or alpha-Synuclein aggregation may not benefit from this research.
What this research studies
This research focuses on developing a novel gene therapy tool called Autophagon, designed to target and clear toxic proteins associated with neurodegenerative diseases like Alzheimer's and Parkinson's. By using viral vectors, the therapy aims to deliver a synthetic gene fragment that helps sequester harmful aggregates of Amyloid-β and alpha-Synuclein, facilitating their removal through the body's natural autophagy process. The approach will be tested in laboratory settings using advanced neuronal cultures derived from stem cells with specific genetic mutations linked to these diseases.
Why it matters
Potential benefit: If successful, this research could lead to effective therapies that significantly alleviate symptoms and slow the progression of Alzheimer's and Parkinson's diseases.
How similar studies have gone: While the approach of using gene therapy for neurodegenerative diseases is innovative, similar strategies targeting protein clearance have shown promise in preliminary studies.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Yakoub, Abraam M. — Brigham and Women's Hospital
- Study coordinator: Yakoub, Abraam M.
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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.