Using sound waves to improve gene therapies for diseases like sickle cell disease
Nanoscience-Inspired Acoustofluidic Assembly Lines for Gene and Cellular Therapies
This study is exploring new ways to improve gene therapy for sickle cell disease by using sound waves to help deliver gene-editing tools into cells more safely and effectively.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project ID | NIH-10693313 on NIH RePORTER |
What this research studies
This research focuses on developing innovative methods to enhance gene therapies using nanotechnology and acoustofluidics. By employing sound waves to temporarily make cell membranes porous, the project aims to facilitate the efficient delivery of gene-editing tools, specifically CRISPR/Cas9, into cells. The approach seeks to overcome current limitations in gene therapy delivery methods, making it safer and more effective for clinical applications. The initial target for this technology is sickle cell disease, a genetic condition caused by a specific mutation.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with sickle cell disease or other genetic conditions that could benefit from advanced gene-editing therapies.
Not a fit: Patients with conditions not related to genetic mutations or those who do not have access to the required technology may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and accessible gene therapies for patients with genetic disorders.
How similar studies have performed: Other research has shown promise in using acoustofluidics and nanotechnology for gene delivery, indicating potential for success in this novel approach.
Where this research is happening
Los Angeles, United States
- University of California Los Angeles — Los Angeles, United States (Active)
Researchers
- Principal investigator: Jonas, Steven John — University of California Los Angeles
- Study coordinator: Jonas, Steven John
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.