Gene therapy to treat idiopathic pulmonary fibrosis by reducing harmful signaling in the lungs
Adeno-associated virus-mediated SGPL1 gene therapy as a first-in-class treatment for idiopathic pulmonary fibrosis that acts by reducing sphingosine-1-phosphate and downstream profibrotic signaling
This study is testing a new gene therapy that uses a virus to help your body break down a harmful substance linked to lung inflammation in people with idiopathic pulmonary fibrosis (IPF), with the hope of improving lung function and managing this tough condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11200757 on NIH RePORTER |
What this research studies
This research investigates a novel gene therapy approach for idiopathic pulmonary fibrosis (IPF), a serious lung disease that leads to progressive lung dysfunction. The therapy uses an adeno-associated virus to deliver the SGPL1 gene, which helps break down a harmful lipid that contributes to lung inflammation and fibrosis. By enhancing the body's ability to degrade sphingosine-1-phosphate, the therapy aims to reduce the fibrotic signaling pathways that worsen IPF. Patients may receive this treatment intravenously, potentially offering a new avenue for managing or curing this debilitating condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with idiopathic pulmonary fibrosis, particularly those who are not responding well to existing treatments.
Not a fit: Patients with other forms of lung disease or those who are not diagnosed with idiopathic pulmonary fibrosis may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with idiopathic pulmonary fibrosis, potentially reversing lung damage and improving survival rates.
How similar studies have performed: While targeting sphingosine-1-phosphate has shown promise in other fibrotic diseases, this specific gene therapy approach is novel and has not been extensively tested in the context of idiopathic pulmonary fibrosis.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Saba, Julie D — University of California, San Francisco
- Study coordinator: Saba, Julie D
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.