Developing a new method for delivering gene therapy using mouse models
Official title: Prototype Mouse Models for SEND-Based mRNA Delivery and Technology Development
Funded at University of Nebraska Medical Center · NIH-10804267 · Data updated 2026-11-12
This study is looking at a new way to deliver gene therapy using special particles that can carry healthy genes to the right cells, which could lead to better treatments for genetic diseases.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Nebraska Medical Center NIH-funded |
| Lab location | 1 site (Omaha, United States) |
| Project start | September 18, 2024 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $330,025 |
| NIH project number | 1R01GM149756-01A1 |
| Project ID | NIH-10804267 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic diseases that could be treated through gene therapy.
✕Less likely to help
Patients with conditions not related to genetic disorders may not receive any benefit from this research.
What this research studies
This research investigates a novel approach to gene therapy delivery using a system called SEND, which utilizes a cellular protein to create viral-like particles that can transport healthy gene copies to target cells. By studying this process in mouse models, the researchers aim to overcome the limitations of current gene therapy delivery methods, such as viral vectors and lipid nanoparticles. The project will explore how these SEND particles can effectively deliver genetic material, potentially leading to more reliable treatments for genetic diseases. Patients may benefit from advancements in gene therapy that could arise from this innovative delivery technology.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for patients with genetic disorders.
How similar studies have gone: While the SEND system shows promise based on preliminary results, it represents a novel approach that has not yet been widely tested in clinical settings.
Where this research is happening
-
University of Nebraska Medical Center
Omaha, United States Active
Researchers
- Principal investigator: Gurumurthy, Channabasavaiah — University of Nebraska Medical Center
- Study coordinator: Gurumurthy, Channabasavaiah
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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.