Stem-cell approaches to understand and treat genetic lung diseases
Developing Pluripotent Stem Cells to Model and Treat Lung Disease
This program uses patient-derived stem cells, mini-lung organoids, and gene editing to develop ways to fix and repair genetic airway and alveolar lung disorders.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston University Medical Campus NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11309135 on NIH RePORTER |
What this research studies
Researchers will reprogram patient cells into pluripotent stem cells and grow lung organoids to model how genetic mutations damage airways and alveoli. The team will use gene-editing tools to correct disease-causing mutations in these lab-grown tissues and test whether corrected cells can restore normal function. A shared Gene Editing Core and common stem-cell resources will support multiple linked projects aimed at moving lab findings toward therapy. Over the funding period the investigators plan to translate promising laboratory results into approaches that could lead to future patient trials.
Who could benefit from this research
Good fit: People with known genetic mutations causing airway or alveolar lung disease who could donate cells or be candidates for future clinical trials are the most relevant participants.
Not a fit: Patients whose lung disease is not caused by a known genetic mutation (for example many cases of smoking-related COPD) are unlikely to benefit directly in the near term.
Why it matters
Potential benefit: If successful, this work could enable new regenerative or gene-based therapies that repair damaged airways and alveoli in people with inherited lung diseases.
How similar studies have performed: Lab studies using iPSCs, organoids, and CRISPR-based gene correction have shown promising preclinical results, but translating these approaches into proven patient treatments is still early and limited.
Where this research is happening
Boston, United States
- Boston University Medical Campus — Boston, United States (Active)
Researchers
- Principal investigator: Kotton, Darrell N. — Boston University Medical Campus
- Study coordinator: Kotton, Darrell N.
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.