Targeted delivery of genome editing tools to lung cells
Official title: Targeted Delivery of Cas9 to Lung Epithelial Cells using Enveloped Delivery Vehicles
Funded at J. David Gladstone Institutes · NIH-11061923 · Data updated 2026-11-12
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 start | April 1, 2024 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $283,500 |
| NIH project number | 5R21HL173710-02 |
| Project ID | NIH-11061923 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients suffering from pulmonary diseases, particularly those involving lung epithelial cells.
✕Less likely to help
Patients with conditions unrelated to lung epithelial cells or those who do not require genome editing therapies may not benefit from this research.
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.
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 gone: 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
-
J. David Gladstone Institutes
San Francisco, United States Active
Researchers
- Principal investigator: Doudna, Jennifer a — J. David Gladstone Institutes
- Study coordinator: Doudna, Jennifer a
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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.