Using non-viral nanovectors to deliver gene therapy for Alzheimer's disease
Official title: Brain-Wide Genome Editing Enabled by Intravenously Administered Non-Viral Nanovectors As a Potential Therapy for Alzheimer’s Disease
Funded at University of Wisconsin-Madison · NIH-10630541 · Data updated 2026-11-12
This study is exploring a new way to treat Alzheimer's disease by using tiny particles to deliver gene-editing tools directly to the brain, which could help tackle the underlying issues of the disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Wisconsin-Madison NIH-funded |
| Lab location | 1 site (Madison, United States) |
| Project start | May 15, 2023 |
| Project end | April 30, 2026 |
| Funding year | 2023 |
| Award (this year) | $1,860,250 |
| NIH project number | 1RF1AG081212-01 |
| Project ID | NIH-10630541 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are older adults diagnosed with Alzheimer's disease or those at high risk for developing it.
✕Less likely to help
Patients with early-stage Alzheimer's or those with other forms of dementia unrelated to Alzheimer's may not benefit from this research.
What this research studies
This research aims to develop a new method for treating Alzheimer's disease by using non-viral nanovectors to deliver CRISPR genome editing tools directly to the brain. The approach focuses on overcoming the challenges posed by the blood-brain barrier, which has made it difficult to effectively administer therapies for neurodegenerative diseases. By engineering silica nanocapsules that can bypass this barrier, the research seeks to enable widespread gene editing throughout the brain, potentially addressing the root causes of Alzheimer's. The effectiveness and safety of this method will be evaluated using a specialized mouse model that mimics Alzheimer's disease.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking therapy that slows or prevents the progression of Alzheimer's disease.
How similar studies have gone: While gene therapy for Alzheimer's is a novel approach, similar strategies using CRISPR technology have shown promise in other neurodegenerative diseases.
Where this research is happening
-
University of Wisconsin-Madison
Madison, United States Active
Researchers
- Principal investigator: Gong, Shaoqin - — University of Wisconsin-Madison
- Study coordinator: Gong, Shaoqin -
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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.