Improving gene therapy for genetic diseases using AAV vectors
Preserving Genome Integrity In AAV-Mediated Gene Therapy
This study is looking at ways to make gene therapy safer and more effective for people with genetic disorders like hemophilia by using smaller doses of a special virus, so it can deliver the needed genes without causing side effects.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11019775 on NIH RePORTER |
What this research studies
This research focuses on enhancing the safety and effectiveness of adeno-associated virus (AAV) vectors used in gene therapy for genetic disorders like hemophilia. The team aims to reduce the required doses of AAV vectors to minimize potential liver toxicity and immune responses that can occur with high doses. By investigating the integrity of the AAV genomes and their behavior in the body, the researchers hope to develop strategies that ensure more reliable gene delivery and expression. This work involves both laboratory studies and animal models to better understand how to optimize AAV vector use.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders, particularly hemophilia, who may benefit from gene therapy.
Not a fit: Patients with conditions not related to genetic disorders or those who do not respond to AAV-mediated therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with genetic diseases.
How similar studies have performed: Previous research has shown promise in optimizing AAV vector delivery, but this specific approach to preserving genome integrity is novel and untested.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Sabatino, Denise E — Children's Hosp of Philadelphia
- Study coordinator: Sabatino, Denise E
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.