Investigating how a specific protein helps adeno-associated virus vectors deliver genes effectively
Official title: Studies of the KIAA0319L-mediated productive transduction pathways of rAAV vectors
Funded at University of Kansas Medical Center · NIH-10997358 · Data updated 2026-11-12
This study is looking at how a special virus used in gene therapy gets into cells and delivers helpful genes, focusing on a protein that helps this process, with the goal of making gene therapies work better for people with different health conditions.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Medical Center NIH-funded |
| Lab location | 1 site (Kansas City, United States) |
| Project start | December 11, 2023 |
| Project end | October 31, 2025 |
| Funding year | 2025 |
| Award (this year) | $225,525 |
| NIH project number | 5R21AI180416-02 |
| Project ID | NIH-10997358 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include patients with genetic conditions that could be treated with rAAV-based gene therapies, such as inherited blindness or spinal muscular atrophy.
✕Less likely to help
Patients with conditions that are not targeted by rAAV-based therapies may not receive any benefit from this research.
What this research studies
This research focuses on understanding the mechanisms by which recombinant adeno-associated virus (rAAV) vectors, which are used in gene therapy, enter cells and deliver genetic material. The study specifically examines the role of a protein called KIAA0319L, which has been identified as a receptor that facilitates the binding and entry of these vectors into cells. By exploring the interactions between rAAV vectors and KIAA0319L, the research aims to overcome existing barriers to effective gene delivery, potentially improving the outcomes of gene therapies for various conditions.
Why it matters
Potential benefit: If successful, this research could enhance the effectiveness of gene therapies, leading to better treatment options for patients with genetic disorders.
How similar studies have gone: Previous research has shown promising results with rAAV vectors in gene therapy, indicating that this approach has potential for success.
Where this research is happening
-
University of Kansas Medical Center
Kansas City, United States Active
Researchers
- Principal investigator: Qiu, Jianming — University of Kansas Medical Center
- Study coordinator: Qiu, Jianming
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.