Using focused ultrasound to deliver gene-editing tools to the brain for neurodegenerative diseases
Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders
This study is exploring a new way to use sound waves to deliver gene-editing tools directly to the brain, which could help improve treatments for conditions like Alzheimer's and Parkinson's by making them more targeted and effective.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10619032 on NIH RePORTER |
What this research studies
This research investigates a novel method to deliver gene-editing elements directly to the brain using focused ultrasound technology. By combining this non-invasive approach with specialized delivery systems, the study aims to enhance the efficiency of CRISPR-based treatments for neurodegenerative disorders like Alzheimer's and Parkinson's diseases. Patients may benefit from improved targeting of therapeutic agents to specific brain regions, potentially leading to better treatment outcomes. The research involves both animal models and human applications to evaluate the effectiveness of this delivery method.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Alzheimer's or Parkinson's diseases who are seeking innovative treatment options.
Not a fit: Patients with neurodegenerative disorders not related to Alzheimer's or Parkinson's may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for neurodegenerative diseases, potentially slowing or reversing disease progression.
How similar studies have performed: Previous studies have shown promise in using focused ultrasound for drug delivery to the brain, indicating potential success for this novel approach.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Leong, Kam W — Columbia University Health Sciences
- Study coordinator: Leong, Kam W
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.