NIH-funded research R01 grant

Developing a platform to test drugs for fatty liver disease based on genetic variants

Official title: 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

Funded at University of Pittsburgh at Pittsburgh · NIH-11019799 · Data updated 2026-11-12

This study is looking at fatty liver disease and aims to create a mini-liver model to help find new treatments, so people with this condition can learn more about how their genes affect their health and potentially benefit from new targeted therapies.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Pittsburgh at Pittsburgh
NIH-funded
Lab location1 site (Pittsburgh, United States)
Project startFebruary 1, 2024
Project endNovember 30, 2028
Funding year2025
Award (this year)$598,763
NIH project number5R01DK135606-02
Project IDNIH-11019799 on NIH RePORTER

Who could benefit from this research

Could help

Ideal candidates for this research are individuals diagnosed with metabolic dysfunction associated fatty liver disease, particularly those with known genetic variants linked to the condition.

Less likely to help

Patients without metabolic dysfunction associated fatty liver disease or those with unrelated liver conditions may not benefit from this research.

What this research studies

This research focuses on metabolic dysfunction associated fatty liver disease (MAFLD), a significant public health issue with no approved treatments. The team is creating a human liver microphysiology system that mimics the liver's environment, allowing for the study of various genetic variants linked to MAFLD. By using advanced computational methods and genome-edited stem cells, the researchers aim to identify and test potential drugs that could effectively treat this condition. Patients may benefit from insights into how specific genetic factors influence their disease and the development of targeted therapies.

Why it matters

Potential benefit: If successful, this research could lead to new or repurposed drugs that effectively treat fatty liver disease based on individual genetic profiles.

How similar studies have gone: Other research has shown promise in using similar biomimetic systems and computational approaches to develop targeted therapies for complex diseases.

Where this research is happening

  • 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.

Related research

Other NIH-funded projects on the same topics.

  • Role of fat droplets in liver fat and inflammatory signals in NAFLD

    A study at Johns Hopkins will explore how fat droplets in the liver influence molecules that drive inflammation and progression of non-alcoholic fatty liver disease (NAFLD) in adults.

    NIH research Johns Hopkins University · Baltimore, United States · NIH-11623263

  • Them1 Inhibitors to Help Treat Fatty Liver Disease

    Aims to develop and test small molecule drugs that block Them1 to help people with non-alcoholic fatty liver disease (NAFLD).

    NIH research Cedars-Sinai Medical Center · Los Angeles, United States · NIH-11519341

  • How the TET1 gene affects non-alcoholic fatty liver disease

    Researchers are looking at whether changes in the TET1 gene and DNA 'tagging' (methylation) play a role in adult non-alcoholic fatty liver disease.

    NIH research State University New York Stony Brook · Stony Brook, United States · NIH-11447065

  • Hepatocellular Genetic Clues for Fatty Liver in Hispanic Adults

    A large NIH grant helps researchers study why fatty liver develops in Hispanic adults and how genetics interact with lifestyle to affect risk.

    NIH research University of Texas Rio Grande Valley · Edinburg, United States · NIH-11421441

  • Toxic fats, vitamin B12 loss, and AHR may drive fatty liver disease

    This grant looks at how certain environmental chemicals may trigger liver damage linked to fatty liver disease and how a vitamin found in the blood could be affected.

    NIH research Michigan State University · East Lansing, United States · NIH-11398883

  • Glutamine and T cells in fatty liver disease

    A study explores how liver glutamine levels affect inflammatory CD4+ T cells in fatty liver disease to find potential new ways to reduce liver inflammation.

    NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11388863

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.