Restoring retinal connections to protect sight
Synapse rescue and neuroprotection in the retina
A gene therapy delivering the protein NGL2 aims to rebuild and protect the light-sensing connections in the retina for people with inherited retinal degenerations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Washington University NIH-funded |
| Lab location | 1 site (Saint Louis, United States) |
| Project ID | NIH-11242057 on NIH RePORTER |
What this research studies
Researchers are using a viral vector to deliver NGL2, a protein that helps keep rod photoreceptor synapses intact, to see if those connections can be rebuilt in adult tissue. In lab and animal models they have already restored synapse numbers and produced extra synapses, and they will now test how well NGL2 delivery slows photoreceptor loss and preserves vision across different inherited retinal degeneration models. The team will compare disease progression with and without NGL2, determine optimal timing for treatment, and check whether the approach works for IRDs with different genetic causes. Much of the current work is in mice but is intended to guide development of future human therapies.
Who could benefit from this research
Good fit: Adults with inherited retinal degenerations that affect rod photoreceptors, especially those earlier in the course of disease before extensive photoreceptor loss, would be the most likely candidates.
Not a fit: People with very advanced disease who have already lost most photoreceptors or those whose vision loss is due to unrelated eye conditions are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, NGL2 gene therapy could slow or prevent vision loss in some inherited retinal degenerations by rebuilding synapses and protecting photoreceptors.
How similar studies have performed: AAV gene therapies have helped certain inherited retinal diseases in humans, but targeting synapse-maintaining proteins like NGL2 is a newer strategy with promising animal results but no clinical proof yet.
Where this research is happening
Saint Louis, United States
- Washington University — Saint Louis, United States (Active)
Researchers
- Principal investigator: Kerschensteiner, Daniel — Washington University
- Study coordinator: Kerschensteiner, Daniel
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.