Finding new treatment targets for fatty liver (NASH) using people with natural gene knockouts
Discovery of novel therapeutic targets for NASH through deep phenotyping of human knockouts and mechanistic studies
Researchers are using genetic data from people—including those who naturally lack certain genes—to find new drug targets that could help people with fatty liver disease (NASH).
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11141602 on NIH RePORTER |
What this research studies
This project uses genetic and health data from hundreds of thousands of people, including a large Pakistani cohort with many 'human knockouts' and thousands of patients with imaging-confirmed fatty liver. Researchers will link rare and common gene variants to liver enzyme levels and confirmed NAFLD/NASH to spot genes that raise or lower disease risk. They will examine people who naturally lack specific genes to see whether that protects against or worsens liver disease, and then run laboratory studies to understand the underlying biology. The goal is to identify genes or pathways that could become targets for new medicines to treat or prevent NASH.
Who could benefit from this research
Good fit: Adults with confirmed NAFLD or NASH—especially those willing to share imaging records, blood samples, or genetic consent—are the most relevant candidates to participate.
Not a fit: People without fatty liver disease or whose liver disease is chiefly due to alcohol or other non-metabolic causes are less likely to benefit directly from this work.
Why it matters
Potential benefit: If successful, this work could point to new drug targets that prevent or treat NASH and reduce liver scarring.
How similar studies have performed: Previous genetic studies have found some risk genes for NAFLD/NASH, but using large numbers of human gene knockouts for therapeutic target discovery is a newer and less-tested approach.
Where this research is happening
New York, United States
- Columbia University Health Sciences — New York, United States (Active)
Researchers
- Principal investigator: Tabas, Ira a — Columbia University Health Sciences
- Study coordinator: Tabas, Ira 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.