Gene therapy to protect vision in children with lysosomal storage diseases
Gene therapy for preserving the visual system in lysosomal storage diseases
This study is testing a new gene therapy that aims to help children with CLN2 disease by protecting their eyesight and brain health with just one treatment, making it easier for families by reducing the need for frequent hospital visits.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Missouri-Columbia NIH-funded |
| Lab location | 1 site (Columbia, United States) |
| Project ID | NIH-10808201 on NIH RePORTER |
What this research studies
This research investigates a gene therapy approach aimed at preventing vision loss and central nervous system degeneration in children affected by CLN2 neuronal ceroid lipofuscinosis. Using a canine model, the study will evaluate the safety and effectiveness of a one-time treatment that delivers gene therapy directly to the eye and cerebrospinal fluid. The goal is to preserve functional vision and neurological health, which are severely impacted by this genetic disorder. If successful, this therapy could provide a less invasive alternative to current treatments that require frequent hospital visits.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with CLN2 neuronal ceroid lipofuscinosis who are experiencing vision loss.
Not a fit: Patients with other forms of lysosomal storage diseases or those not affected by CLN2 disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that preserves vision and neurological function in children with CLN2 disease.
How similar studies have performed: Previous research has shown success with gene therapy approaches in similar conditions, indicating potential for this novel treatment.
Where this research is happening
Columbia, United States
- University of Missouri-Columbia — Columbia, United States (Active)
Researchers
- Principal investigator: Katz, Martin L — University of Missouri-Columbia
- Study coordinator: Katz, Martin L
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.