Gene therapy for treating a hereditary brain disease in cats
Intracerebroventricular cyclodextrin and AAV-mediated gene therapy forglobal CNS disease correction in feline NPC1 disease
This study is looking for a better way to treat cats with Niemann-Pick disease type C1 by using a special gene therapy that delivers the missing gene directly to their brains, aiming to make treatment easier and help these cats live happier, longer lives.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project ID | NIH-11136461 on NIH RePORTER |
What this research studies
This research focuses on Niemann-Pick disease type C1 (NPC1 disease), a genetic disorder that leads to severe neurological symptoms in cats. The study aims to develop a safer and more effective treatment by using a gene therapy approach with an adeno-associated virus (AAV) vector to deliver the NPC1 gene directly to the brain. This method seeks to avoid the need for frequent invasive injections and reduce side effects associated with current treatments. By targeting specific brain regions, the research hopes to improve the quality of life and extend survival for affected cats.
Who could benefit from this research
Good fit: Ideal candidates for this research are cats diagnosed with Niemann-Pick disease type C1.
Not a fit: Cats with other unrelated neurological disorders or those not diagnosed with NPC1 disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that alleviates symptoms and improves the lifespan of cats suffering from NPC1 disease.
How similar studies have performed: Previous studies have shown promise in using AAV vectors for gene therapy in other genetic disorders, indicating potential success for this novel approach.
Where this research is happening
Gainesville, United States
- University of Florida — Gainesville, United States (Active)
Researchers
- Principal investigator: Vite, Charles H — University of Florida
- Study coordinator: Vite, Charles H
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.