Gene therapy to reduce amyloid beta in Alzheimer's disease
Gene therapy for Alzheimer's disease using virally delivered Aβ variants
This study is testing a new gene therapy for Alzheimer's disease that uses a special virus to deliver helpful proteins to the brain, aiming to stop harmful proteins from building up and potentially making things better for people with Alzheimer's.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-10883953 on NIH RePORTER |
What this research studies
This research investigates a novel gene therapy approach for Alzheimer's disease by using adeno-associated virus (AAV) vectors to deliver specific variants of amyloid beta (Aβ) peptides. These peptides are designed to prevent the aggregation of harmful Aβ proteins and promote the disassembly of existing fibrils, potentially reducing toxicity in the brain. The therapy aims to express these peptides in the brain, where they can act directly on the amyloid plaques associated with Alzheimer's. The study will explore the mechanisms of how these variants interact with the normal Aβ proteins and assess their effectiveness in animal models before considering human applications.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults aged 21 and older who are diagnosed with Alzheimer's disease or exhibit early signs of cognitive decline.
Not a fit: Patients with other forms of dementia unrelated to amyloid beta pathology may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment that significantly slows the progression of Alzheimer's disease by reducing toxic amyloid beta levels in the brain.
How similar studies have performed: Previous research has shown promising results with similar gene therapy approaches targeting amyloid beta, indicating potential for success in this novel application.
Where this research is happening
Houston, United States
- Baylor College of Medicine — Houston, United States (Active)
Researchers
- Principal investigator: Jankowsky, Joanna L — Baylor College of Medicine
- Study coordinator: Jankowsky, Joanna L
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.