New treatments for liver disease caused by alpha-1 antitrypsin deficiency
Novel therapies that target mitochondrial dysfunction for treatment of a1-antitrypsin deficiency liver disease
This study is looking for new ways to treat liver disease caused by alpha-1 antitrypsin deficiency, focusing on how problems in cell energy can harm the liver, with the hope of finding better treatment options for patients beyond just liver transplants.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project ID | NIH-10992164 on NIH RePORTER |
What this research studies
This research focuses on developing novel therapies to address liver disease resulting from alpha-1 antitrypsin deficiency (ATD). It investigates the role of mitochondrial dysfunction in the progression of liver damage associated with this genetic condition. By understanding how misfolded proteins accumulate in liver cells and lead to cell damage, the research aims to identify potential therapeutic targets. Patients may benefit from new treatment options beyond liver transplantation, which is currently the only available solution.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with alpha-1 antitrypsin deficiency, particularly those experiencing liver-related complications.
Not a fit: Patients without alpha-1 antitrypsin deficiency or those who do not exhibit liver disease symptoms may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide new treatment options for patients with alpha-1 antitrypsin deficiency liver disease, potentially improving their quality of life and health outcomes.
How similar studies have performed: While there has been limited research specifically targeting mitochondrial dysfunction in alpha-1 antitrypsin deficiency, similar approaches in other liver diseases have shown promise, indicating potential for success.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Rudnick, David a — Washington University
- Study coordinator: Rudnick, David a
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.