Understanding how urea cycle disorders affect liver metabolism

DISSECTING THE LINK BETWEEN UREAGENESIS AND HEPATIC GLYCOGEN METABOLISM

NIH-funded research Baylor College of Medicine · NIH-11014361

This study is looking at how urea cycle disorders can affect liver health, especially why some patients still develop liver problems even with better treatments, and it aims to find new ways to help prevent these issues for people with these conditions.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionBaylor College of Medicine NIH-funded
Lab location1 site (Houston, United States)
Project IDNIH-11014361 on NIH RePORTER

What this research studies

This research investigates the relationship between urea cycle disorders (UCDs) and liver metabolism, particularly focusing on how these disorders can lead to chronic liver disease despite improved treatments. The study aims to explore the mechanisms behind excess glycogen accumulation in the liver, which is a common issue in patients with UCDs like argininosuccinate lyase deficiency. By using mouse models, researchers will examine how disruptions in energy metabolism due to urea cycle dysfunction contribute to liver disease. The goal is to identify potential therapeutic strategies to prevent liver complications in affected patients.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with urea cycle disorders, particularly those experiencing liver complications.

Not a fit: Patients without urea cycle disorders or those whose liver disease is unrelated to metabolic dysfunction may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new treatments that specifically target liver disease in patients with urea cycle disorders.

How similar studies have performed: While there have been studies on urea cycle disorders, this research aims to explore novel mechanisms that have not been extensively tested in relation to liver disease.

Where this research is happening

Houston, United States

Researchers

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 below.
Conditions Argininosuccinate Synthase Deficiency DiseaseArgininosuccinic Acid Synthase Deficiency DiseaseArgininosuccinic Acid Synthetase Deficiency Disease
Last reviewed 2026-06-13 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.