Therapies for newborns with alveolar capillary dysplasia
Development of novel therapeutic approaches for treatment of Alveolar Capillary Dysplasia
Trying genetic and molecular approaches to fix FOXF1-related lung problems in newborns with alveolar capillary dysplasia (ACDMPV).
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Arizona NIH-funded |
| Lab location | 1 site (Tucson, United States) |
| Project ID | NIH-11220915 on NIH RePORTER |
What this research studies
Researchers are studying the FOXF1 gene and its regulatory enhancers using samples from human lungs and specially engineered mice to guide new treatments. They use single-cell RNA and ATAC sequencing to pinpoint faulty enhancer elements and employ CRISPR tools to create and study a mouse model of the type 3 enhancer loss seen in many patients. The team will test molecular or gene-based interventions in these preclinical models to see if lung development and blood vessel formation can be restored. If successful in animals, these approaches would inform future therapies for affected newborns.
Who could benefit from this research
Good fit: Newborns diagnosed with ACDMPV, particularly those with FOXF1 mutations or loss of the FEL1 enhancer (type 3), would be the main candidates for future trials or therapies.
Not a fit: Patients whose lung disease is not caused by FOXF1-related defects, or older children and adults without that specific genetic cause, may not benefit from these approaches.
Why it matters
Potential benefit: Could lead to therapies that prevent fatal respiratory failure in newborns with ACDMPV.
How similar studies have performed: Related preclinical work using mouse models and gene-based methods has shown promise, but there are currently no proven treatments for ACDMPV in humans.
Where this research is happening
Tucson, United States
- University of Arizona — Tucson, United States (Active)
Researchers
- Principal investigator: Kalinichenko, Vladimir — University of Arizona
- Study coordinator: Kalinichenko, Vladimir
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.