Targeted delivery of genome editing tools to lung cells
Targeted Delivery of Cas9 to Lung Epithelial Cells using Enveloped Delivery Vehicles
This study is testing a new way to deliver gene-editing tools directly to lung cells to help treat lung diseases, using specially designed particles that can safely carry the treatment right where it's needed.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | J. David Gladstone Institutes NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11061923 on NIH RePORTER |
What this research studies
This research focuses on developing a new method to deliver genome editing tools specifically to lung epithelial cells, which are crucial in treating pulmonary diseases. The approach involves engineering virus-like particles, known as Enveloped Delivery Vehicles (EDVs), that can carry Cas9 proteins directly to the targeted cells. By enhancing the specificity of delivery, this research aims to minimize off-target effects and improve the safety and effectiveness of CRISPR-Cas9 treatments for lung conditions. The study will explore how these EDVs can encapsulate Cas9 and its guide RNA to ensure precise targeting.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients suffering from pulmonary diseases, particularly those involving lung epithelial cells.
Not a fit: Patients with conditions unrelated to lung epithelial cells or those who do not require genome editing therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer treatments for patients with lung diseases by enabling precise genome editing.
How similar studies have performed: Other research has shown promise in using engineered virus-like particles for targeted delivery, indicating potential success for this novel approach.
Where this research is happening
San Francisco, United States
- J. David Gladstone Institutes — San Francisco, United States (Active)
Researchers
- Principal investigator: Doudna, Jennifer a — J. David Gladstone Institutes
- Study coordinator: Doudna, Jennifer a
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.