Human lab-grown livers to find medicines for metabolic fatty liver disease
Implementing A QSP Platform to Predict and Test Drugs for Metabolic Associated Fatty Liver Disease Genetic Variants in an iPSC-cell Based Human Biomimetic Liver Microphysiology System
This project uses computer models and tiny human liver tissues grown from stem cells to find medicines that could help people with metabolic fatty liver disease linked to specific genes.
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-11290358 on NIH RePORTER |
What this research studies
From a patient view, researchers will build tiny, blood‑flowing human livers in the lab using stem cells and the exact genetic changes linked to fatty liver disease. They'll combine those lab models with computer simulations to predict which existing or new drugs—or drug combinations—might fix disease features caused by different gene variants. The team will edit genes in the stem‑cell derived liver cells to mimic both risk and protective human variants and watch how the tissues respond at the molecular and functional level. The goal is to point to drug candidates and explain how certain gene changes protect or harm the liver, which could guide future clinical trials.
Who could benefit from this research
Good fit: People with metabolic dysfunction‑associated fatty liver disease (MAFLD), especially those known to carry genetic variants such as PNPLA3, TM6SF2, MBOAT7, HSD17B13, or MTARC1, are the most likely future candidates to benefit from follow-up trials based on this work.
Not a fit: People without metabolic fatty liver disease or those with very advanced, irreversible liver failure are unlikely to benefit directly from this laboratory-focused project.
Why it matters
Potential benefit: If successful, this work could identify new or repurposed medicines tailored to genetic types of metabolic fatty liver disease and speed the path to patient trials.
How similar studies have performed: Related lab‑grown liver models and genetic studies have produced useful biological clues, but combining genome‑edited human iPSC livers with quantitative systems pharmacology to pick drugs is a relatively new and emerging approach.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Miedel, Mark T. — University of Pittsburgh at Pittsburgh
- Study coordinator: Miedel, Mark T.
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.