Understanding why a rare pediatric lung disease happens and how to treat it
Official title: Deciphering the Molecular Mechanism of Pediatric Interstitial Lung Disease to Advance Targeted Therapies
Funded at University of Pennsylvania · NIH-11412423 · Data updated 2026-11-12
Aims to uncover the molecular causes of pediatric interstitial lung disease and use that knowledge to develop targeted therapies for children affected by this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 15, 2025 |
| Project end | June 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $601,596 |
| NIH project number | 5R01HL176680-02 |
| Project ID | NIH-11412423 on NIH RePORTER |
Who could benefit from this research
✓Could help
Pediatric patients with or at risk for ChILD due to SFTPC mutations or related alveolar epithelial dysfunction may benefit from advances in this research.
✕Less likely to help
Children without SFTPC mutations or without ChILD-related alveolar epithelial dysfunction may not directly benefit.
What this research studies
This project uses patient-derived cells and a mouse model to study how specific genetic mutations in SFTPC affect lung development and lead to disease. By examining the early events in alveolar type 2 cells, it seeks to map the pathways that go wrong and test potential therapies in preclinical systems. The team has already created a relevant in vivo model and iPSC-derived airway cells from a patient to explore disease mechanisms and treatment options. The findings could point to gene-based or other targeted interventions that may slow or halt progression in affected children.
Why it matters
Potential benefit: If successful, the work could lead to targeted therapies that improve breathing and slow disease progression in children with ChILD.
How similar studies have gone: Previous models and patient-derived cells have shown that SFTPC-related ChILD requires precise alveolar type 2 cell biology, and this study builds on those insights with advanced preclinical models.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Katzen, Jeremy Binder — University of Pennsylvania
- Study coordinator: Katzen, Jeremy Binder
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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.