Investigating a transporter that affects a toxic compound in certain genetic diseases
The ABCG2 transporter in protoporphyrin IX disposition: from toxicity to therapy
This study is looking for new ways to help people with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) by creating treatments that can reduce the harmful effects of a substance called protoporphyrin IX, which can cause serious skin and liver issues.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-11083696 on NIH RePORTER |
What this research studies
This research focuses on two genetic conditions, erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), which cause harmful effects due to a compound called protoporphyrin IX (PPIX). The researchers aim to develop new treatments by targeting a specific protein, ABCG2, that influences how PPIX is processed in the body. By creating inhibitors of ABCG2, they hope to reduce the toxic effects of PPIX, which can lead to severe skin and liver problems. The study involves synthesizing and testing these inhibitors for their effectiveness and safety in managing the toxicities associated with these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with erythropoietic protoporphyria or X-linked protoporphyria.
Not a fit: Patients without these specific genetic conditions or those not affected by PPIX-mediated toxicities may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly reduce the harmful effects of PPIX in patients with EPP and XLP.
How similar studies have performed: Previous research has shown promise in targeting similar transporters for managing toxicities, suggesting a potential for success in this novel approach.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Ma, Xiaochao — University of Pittsburgh at Pittsburgh
- Study coordinator: Ma, Xiaochao
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.