Using lipid nanoparticles to target the fetal lung for treatment of congenital lung diseases
Ionizable Lipid Nanoparticles for Fetal Lung Targeting
This study is exploring a new way to treat lung diseases in unborn babies by using tiny particles to deliver helpful genetic material directly to their lungs, which could fix problems before they are born and help them breathe better after birth.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11098671 on NIH RePORTER |
What this research studies
This research focuses on developing a new method to treat congenital lung diseases in fetuses, such as alpha-1 antitrypsin deficiency. By utilizing ionizable lipid nanoparticles, the study aims to deliver genetic material directly to the fetal lungs, potentially correcting genetic defects before birth. This approach leverages advancements in gene editing technologies, allowing for early intervention that could improve lung function and reduce complications after birth. The research will be conducted using animal models to assess the effectiveness and safety of this innovative delivery system.
Who could benefit from this research
Good fit: Ideal candidates for this research are fetuses diagnosed with congenital lung diseases, particularly those with alpha-1 antitrypsin deficiency.
Not a fit: Patients who are already born and have established lung conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking therapies that correct congenital lung diseases before birth, significantly improving outcomes for affected infants.
How similar studies have performed: While gene editing in utero is a novel approach, previous studies using viral vectors have shown promise, indicating potential for success with this new non-viral method.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Palanki, Rohan — University of Pennsylvania
- Study coordinator: Palanki, Rohan
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.