Targeting alveolar cell problems caused by SFTPC gene changes in pulmonary fibrosis
Alveolar Epithelial Cell Dysfunction in Pulmonary Fibrosis: Leveraging SFTPC Mutations for Discovery of Molecular and Cellular Targets
This project looks for new ways to fix how gene changes in SFTPC make alveolar cells malfunction in people with pulmonary fibrosis so new treatments can be developed.
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 ID | NIH-11328669 on NIH RePORTER |
What this research studies
From my perspective as a patient, the team studies how rare changes in the SFTPC (surfactant protein C) gene make lung alveolar type 2 cells behave abnormally and drive scarring in idiopathic pulmonary fibrosis (IPF). They use lab-grown cells and animal models that mimic the SFTPC mutations seen in some patients, and they examine human lung tissue and patient-derived samples to connect lab findings to real disease. The researchers aim to identify specific molecular and cellular pathways that go wrong in these mutated cells and then test interventions that restore normal cell quality control. Their approach links patient genetics, cell biology, and preclinical testing to find targets that could be translated into therapies.
Who could benefit from this research
Good fit: Ideal candidates are people diagnosed with idiopathic pulmonary fibrosis, especially those known to carry or suspected to carry pathogenic SFTPC gene variants or with a family history of PF.
Not a fit: People without pulmonary fibrosis or whose disease is driven by unrelated mechanisms are unlikely to benefit directly from this project in the near term.
Why it matters
Potential benefit: If successful, this work could point to targeted treatments that slow or prevent lung scarring by correcting alveolar cell dysfunction in people with IPF.
How similar studies have performed: Earlier laboratory and in vivo work from this group provided proof-of-concept that SFTPC mutations disrupt epithelial cell quality control, but therapies directly targeting these mutation-driven mechanisms remain largely novel.
Where this research is happening
Philadelphia, United States
- University of Pennsylvania — Philadelphia, United States (Active)
Researchers
- Principal investigator: Beers, Michael Francis — University of Pennsylvania
- Study coordinator: Beers, Michael Francis
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.