Testing gene therapy for a severe childhood brain disease in sheep.
Characterizing and testing the efficacy of AAV-mediated gene therapy in a sheep model of CLN1 disease.
['FUNDING_R01'] · WASHINGTON UNIVERSITY · NIH-10873266
This study is exploring a new gene therapy to help treat CLN1 disease in children by testing it in sheep to see how well it can safely reach the brain and spinal cord, with the goal of finding better ways to help kids who currently have no effective treatments.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | WASHINGTON UNIVERSITY (nih funded) |
| Locations | 1 site (SAINT LOUIS, UNITED STATES) |
| Trial ID | NIH-10873266 on ClinicalTrials.gov |
What this research studies
This research investigates a gene therapy approach for treating CLN1 disease, a severe neurodegenerative condition that affects children. Using a sheep model that mimics the disease, the team will assess how effectively the therapy can be delivered to the brain and spinal cord, focusing on safety and dosing. The study aims to refine the therapeutic strategy to improve outcomes for affected children, as current treatments are ineffective. By leveraging advanced techniques like CRISPR, the researchers hope to translate their findings into potential clinical applications.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with CLN1 disease or those at risk of developing it.
Not a fit: Patients with other forms of Batten disease or those who do not have CLN1 disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment for CLN1 disease, potentially improving the quality of life and outcomes for affected children.
How similar studies have performed: While gene therapy has shown promise in other models of neurodegenerative diseases, this specific approach in CLN1 disease using a sheep model is relatively novel.
Where this research is happening
SAINT LOUIS, UNITED STATES
- WASHINGTON UNIVERSITY — SAINT LOUIS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: COOPER, JONATHAN D — WASHINGTON UNIVERSITY
- Study coordinator: COOPER, JONATHAN D
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.
Conditions: Batten Disease, Batten-Mayou Disease, Batten-Spielmeyer-Vogt Disease