How BMPR2 gene changes cause blocked lung arteries in pulmonary arterial hypertension
BMPR2 mutations, Neointimal Transformation and Pulmonary Arterial Hypertension
Looking at how inherited BMPR2 gene changes combined with lung inflammation drive pulmonary arterial hypertension in people at risk, to point toward better treatments.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Veterans Admin Palo Alto Health Care Sys NIH-funded |
| Lab location | 1 site (Palo Alto, United States) |
| Project ID | NIH-11131021 on NIH RePORTER |
What this research studies
Researchers use a ‘two‑hit’ rat model that combines a BMPR2 mutation with lung inflammation to recreate the blocked, thickened small pulmonary arteries seen in human PAH. They profile individual lung blood vessel cells using single‑cell RNA sequencing to see which endothelial cells transform into the neointima. The team tests whether blocking TGF‑β signaling can reverse or limit this neointimal transformation in the model. Findings are intended to help guide therapies for veterans and others with BMPR2‑linked PAH.
Who could benefit from this research
Good fit: People with pulmonary arterial hypertension, especially those known to carry BMPR2 mutations or with histories of lung inflammation, are the most relevant population for these results.
Not a fit: People with other forms of pulmonary hypertension not driven by BMPR2 mutations or inflammation may not see direct benefit from these specific findings.
Why it matters
Potential benefit: If successful, this work could point to treatments that prevent or reverse artery blockage in BMPR2‑related PAH, improving symptoms and survival.
How similar studies have performed: Related preclinical work, including TGF‑β blockade in BMPR2 mutant animals, has shown improvement, but clinical benefit in people is not yet proven.
Where this research is happening
Palo Alto, United States
- Veterans Admin Palo Alto Health Care Sys — Palo Alto, United States (Active)
Researchers
- Principal investigator: Nicolls, Mark Robert — Veterans Admin Palo Alto Health Care Sys
- Study coordinator: Nicolls, Mark Robert
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.