Understanding how cellular proteins interact with AAV genomes
Official title: Identifying cellular proteins associated with AAV genomes
Funded at Children's Hosp of Philadelphia · NIH-11140995 · Data updated 2026-11-12
This study is looking at how certain proteins in our cells help a virus used in gene therapy work better, which could lead to improved treatments for patients needing gene therapy.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | July 18, 2024 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $205,330 |
| NIH project number | 5R21AI185720-02 |
| Project ID | NIH-11140995 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients with genetic conditions that could be treated using AAV-based gene therapies.
✕Less likely to help
Patients who do not have genetic disorders or conditions that are targeted by AAV-based therapies may not benefit from this research.
What this research studies
This research investigates the interactions between cellular proteins and adeno-associated virus (AAV) genomes, which are crucial for effective gene therapy. By identifying the cellular factors that facilitate AAV replication and transduction, the study aims to enhance the production and delivery of AAV vectors for gene therapy applications. The researchers will utilize advanced proteomics techniques to analyze these interactions, providing insights that could improve gene therapy outcomes. Patients may benefit from improved AAV-based treatments as a result of this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies using AAV vectors, improving treatment options for patients with genetic disorders.
How similar studies have gone: Previous research has shown success in identifying cellular interactions with viral genomes, suggesting that this approach could yield valuable insights for AAV vector development.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Weitzman, Matthew D. — Children's Hosp of Philadelphia
- Study coordinator: Weitzman, Matthew D.
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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.