Personal immune risk in eye gene therapy
Immune activation and subject-specific risk in ocular gene therapies
Researchers will look for genetic differences and blood markers that predict immune reactions to eye gene therapies for people with inherited retinal disorders.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project ID | NIH-11230337 on NIH RePORTER |
What this research studies
If I'm considering gene therapy for an inherited eye disease, researchers will use mouse models to learn why immune reactions and toxic side effects differ between individuals. They'll test whether immunosuppressive drugs change toxicity after injections into the eye, map genetic differences that increase risk, and measure blood markers that might signal susceptibility. Those blood markers will be analyzed with machine learning to build a way to estimate an individual animal's risk before treatment. Although the experiments use mice, the goal is to develop tests and strategies doctors could use to personalize and make ocular gene therapy safer for patients.
Who could benefit from this research
Good fit: People with inherited retinal disorders (for example Bardet-Biedl syndrome) who are considering or eligible for ocular gene therapy would be the eventual beneficiaries of this research.
Not a fit: People without inherited eye disease or those not undergoing gene therapy are unlikely to directly benefit from these mouse-focused experiments.
Why it matters
Potential benefit: If successful, this work could help doctors predict and reduce immune-related side effects from eye gene therapies, making treatments safer and more individualized.
How similar studies have performed: Immune reactions to gene therapies are a known problem and immunosuppression has shown some benefit, but using genetics and blood biomarkers to predict individual risk is relatively new and largely unproven.
Where this research is happening
Iowa City, United States
- University of Iowa — Iowa City, United States (Active)
Researchers
- Principal investigator: Hsu, Ying — University of Iowa
- Study coordinator: Hsu, Ying
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.