Improving gene therapy for CLN3 disease
Official title: Network modulation to improve gene therapy in CLN3 disease
Funded at Children's Hosp of Philadelphia · NIH-10930953 · Data updated 2026-11-12
This study is looking at ways to improve gene therapy for CLN3 disease, which mainly affects kids and causes serious brain problems, by exploring how the disease disrupts brain function, so we can find better treatments to help those who are struggling with it.
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 start | September 18, 2023 |
| Project end | July 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $487,607 |
| NIH project number | 5R01NS126279-02 |
| Project ID | NIH-10930953 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with CLN3 disease, particularly those experiencing neurological symptoms.
✕Less likely to help
Patients with other lysosomal storage disorders that do not involve CLN3 may not benefit from this specific research.
What this research studies
This research investigates how to enhance gene therapy for CLN3 disease, a condition that primarily affects children and leads to severe neurological symptoms. The approach focuses on understanding the underlying brain network disruptions caused by the disease, using advanced techniques like electrophysiology to assess brain function in animal models. By identifying specific physiological changes in the brain, the researchers aim to develop more effective therapeutic strategies that go beyond simply restoring protein levels. This could lead to better outcomes for patients suffering from this debilitating condition.
Why it matters
Potential benefit: If successful, this research could lead to improved gene therapy treatments that significantly enhance the quality of life for children with CLN3 disease.
How similar studies have gone: While gene therapy has been explored in other lysosomal storage disorders, this specific approach focusing on network modulation in CLN3 disease is relatively novel and has not been extensively tested.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Ahrens-Nicklas, Rebecca Clare — Children's Hosp of Philadelphia
- Study coordinator: Ahrens-Nicklas, Rebecca Clare
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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.