Autophagon gene therapy to help brain cells clear harmful Alzheimer’s and Parkinson’s proteins
Autophagon: an Autophagy-Functionalizing Gene Therapy Tool for Neurodegenerative Diseases
A gene therapy that helps brain cells send toxic protein clumps to their natural recycling system for people with Alzheimer’s or Parkinson’s.
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 ID | NIH-11238545 on NIH RePORTER |
What this research studies
Researchers are creating a synthetic gene tool called Autophagon that tags and shuttles toxic amyloid‑β and α‑synuclein aggregates into neurons' autophagy (cellular cleanup) pathway. They will test Autophagon in lab-grown human neurons and 3D brain organoids made from patient-derived stem cells carrying Alzheimer’s or Parkinson’s mutations. The team will also deliver the tool into mouse brains using viral vectors to see whether it lowers protein aggregates and protects nerve cells. Successful lab and animal results would support further development toward human testing.
Who could benefit from this research
Good fit: Future clinical candidates would likely be people diagnosed with Alzheimer’s disease or Parkinson’s disease, especially those in earlier stages or with genetic forms such as APP/PSEN1 or α‑synuclein mutations.
Not a fit: People without evidence of protein‑aggregation disease, those with very advanced neurodegeneration, or anyone now seeking direct treatment will not benefit from this preclinical work.
Why it matters
Potential benefit: If successful, this approach could lower toxic protein buildup in the brain and potentially slow or prevent cognitive and motor decline in Alzheimer’s and Parkinson’s patients.
How similar studies have performed: Related strategies that boost autophagy have cleared toxic proteins in cell and animal models, but gene therapies targeting aggregates have not yet been proven safe and effective in people.
Where this research is happening
Boston, United States
- 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.
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.