How mutations in the ABCA3 gene affect lung cell function and potential gene therapy solutions
Official title: Impact of ATP-binding cassette A3 (ABCA3) mutations on alveolar type 2 cell function and genetic complementation
Funded at University of Iowa · NIH-10953882 · Data updated 2026-11-12
This study is looking at how changes in the ABCA3 gene affect lung cells that are important for breathing, with the goal of finding better treatments for conditions like neonatal respiratory distress syndrome and pulmonary fibrosis, and it aims to explore gene therapy as a way to fix these issues for patients.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project start | August 1, 2024 |
| Project end | July 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $173,175 |
| NIH project number | 1R03TR004814-01A1 |
| Project ID | NIH-10953882 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants and children diagnosed with respiratory distress syndrome or interstitial lung diseases linked to ABCA3 mutations.
✕Less likely to help
Patients with lung diseases not associated with ABCA3 mutations or those who are adults may not benefit from this research.
What this research studies
This research investigates the impact of mutations in the ABCA3 gene on the function of alveolar type 2 cells, which are crucial for lung health. The study aims to understand how these genetic changes lead to conditions like neonatal respiratory distress syndrome and pulmonary fibrosis. By exploring gene therapy strategies, the researchers hope to restore normal ABCA3 function in lung cells, potentially improving treatment options for affected patients. The approach includes laboratory experiments to assess the biological effects of these mutations and the effectiveness of gene therapy techniques.
Why it matters
Potential benefit: If successful, this research could lead to new gene therapy treatments for severe lung diseases caused by ABCA3 mutations.
How similar studies have gone: Previous research has shown promise in using gene therapy for similar genetic conditions, indicating potential for success in this approach.
Where this research is happening
-
University of Iowa
Iowa City, United States Active
Researchers
- Principal investigator: Mccray, Paul B — University of Iowa
- Study coordinator: Mccray, Paul B
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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.