Using advanced CRISPR technology to edit genes in the brain for Alzheimer's treatment
Official title: Nanoformulated CRISPR Ribonucleoproteins for Ultrasound-Facilitated Brain Gene Editing
Funded at State University of New York at Buffalo · NIH-10727386 · Data updated 2026-11-12
This study is exploring a new way to help people with Alzheimer's by using tiny gene-editing tools and sound waves to deliver treatment directly to the brain, making it safer and more effective for those affected by the disease.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University of New York at Buffalo NIH-funded |
| Lab location | 1 site (Amherst, United States) |
| Project start | July 1, 2023 |
| Project end | June 30, 2026 |
| Funding year | 2023 |
| Award (this year) | $442,000 |
| NIH project number | 1R21NS133635-01 |
| Project ID | NIH-10727386 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Alzheimer's disease or related neurodegenerative disorders.
✕Less likely to help
Patients with early-stage Alzheimer's or those without a confirmed diagnosis may not benefit from this research.
What this research studies
This research investigates a novel approach to treat Alzheimer's disease by utilizing nanoformulated CRISPR ribonucleoproteins combined with ultrasound technology to facilitate gene editing in the brain. The study aims to overcome the challenges of delivering CRISPR components effectively to the brain, which is crucial for addressing neuronal degeneration disorders. By creating a library of lipid-coated CRISPR components and optimizing methods to open the blood-brain barrier, the research seeks to enhance the safety and efficacy of gene therapy for Alzheimer's patients.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients suffering from Alzheimer's disease.
How similar studies have gone: While gene editing technologies are emerging, this specific approach using nanoformulated CRISPR for brain applications is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
-
State University of New York at Buffalo
Amherst, United States Active
Researchers
- Principal investigator: Lao, Yeh-Hsing — State University of New York at Buffalo
- Study coordinator: Lao, Yeh-Hsing
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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.