Long-lasting gene therapy to lower eye pressure for glaucoma
Development of an IOP-lowering gene therapy treatment for glaucoma
A one-time gene therapy injected into the front of the eye that aims to make eye tissues produce a pressure-lowering medicine for people with open-angle or congenital glaucoma.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project ID | NIH-11099767 on NIH RePORTER |
What this research studies
This project is developing a one-time gene therapy delivered with a harmless viral vector into the eye so cornea and outflow pathway cells can be instructed to make prostaglandin F2α, a drug that lowers intraocular pressure. The plan is for treated cells to continuously secrete the drug so patients would not need daily eye drops. Researchers are testing delivery, targeting, and safety in laboratory and animal studies to see how well the approach controls pressure over time. If preclinical results support safety and effect, future clinical trials would invite people with glaucoma to receive the treatment and be followed for vision and eye pressure outcomes.
Who could benefit from this research
Good fit: People with open-angle glaucoma or congenital glaucoma who have elevated intraocular pressure or who struggle with daily eye-drop adherence would be the most likely candidates.
Not a fit: People with angle-closure glaucoma, major corneal scarring, very advanced irreversible optic nerve damage, or eye problems not driven by high pressure are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, a single treatment could provide long-term lowering of eye pressure, reduce reliance on daily eye drops, and lower the risk of glaucoma-related vision loss.
How similar studies have performed: AAV gene therapies have shown success in treating some inherited eye diseases and prostaglandin eye drops are proven to lower pressure, but using AAV to continuously produce prostaglandin in the eye is a novel strategy still at early preclinical stages.
Where this research is happening
Milwaukee, United States
- Medical College of Wisconsin — Milwaukee, United States (Active)
Researchers
- Principal investigator: Lipinski, Daniel Mark — Medical College of Wisconsin
- Study coordinator: Lipinski, Daniel Mark
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.