How the Liver Processes Antisense Oligonucleotide Medicines
Official title: Metabolism of Antisense Oligonucleotides and other Polyanions in Liver
Funded at University of Nebraska Lincoln · NIH-11131213 · Data updated 2026-11-12
This research explores how the liver handles a type of medicine called antisense oligonucleotides, which are used to treat various diseases by targeting specific genes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Nebraska Lincoln NIH-funded |
| Lab location | 1 site (Lincoln, United States) |
| Project start | September 1, 2022 |
| Project end | June 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $292,661 |
| NIH project number | 5R01GM147913-04 |
| Project ID | NIH-11131213 on NIH RePORTER |
Who could benefit from this research
✓Could help
This foundational research does not directly involve patients, but its findings could ultimately benefit individuals receiving or candidates for antisense oligonucleotide therapies for various genetic conditions.
✕Less likely to help
Patients not currently using or considering antisense oligonucleotide therapies would not directly benefit from this specific research.
What this research studies
Antisense oligonucleotides, or ASOs, are small molecules designed to stop disease-causing genes from working by binding to their genetic instructions. While ASOs are effective, we know that the liver naturally takes up many of these medicines, but the exact process isn't fully understood. This project aims to uncover the specific ways the liver's scavenger receptors interact with ASOs, determining if they are broken down or allowed to reach their targets within cells. Understanding these pathways is key to making ASO therapies more efficient and predictable for patients.
Why it matters
Potential benefit: If successful, this work could lead to improved design of ASO medicines, making them more effective and reducing potential side effects by better controlling how they are processed in the body.
How similar studies have gone: While ASO therapies are already in clinical use, this specific research explores novel mechanisms of their metabolism in the liver, building on recent discoveries by the research team.
Where this research is happening
-
University of Nebraska Lincoln
Lincoln, United States Active
Researchers
- Principal investigator: Harris, Edward N — University of Nebraska Lincoln
- Study coordinator: Harris, Edward N
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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.