Correcting genes that raise the risk of age-related macular degeneration
Editing AMD Risk Alleles in Human Cells
['FUNDING_R01'] · UNIVERSITY OF IOWA · NIH-11303255
This project uses precise gene-editing tools to change specific DNA variants tied to AMD in human eye cells to lower future risk for people with high-risk genes.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF IOWA (nih funded) |
| Locations | 1 site (IOWA CITY, UNITED STATES) |
| Trial ID | NIH-11303255 on ClinicalTrials.gov |
What this research studies
Researchers will apply modern base editing and prime editing methods to alter AMD-linked risk alleles at two main sites (ARMS2/HTRA1 on 10q26 and CFH on 1q) in human-derived cells. They will work in lab-grown iPSC-derived retinal pigment epithelium, retinal and choroidal cells, and in donor retinal and choroidal explant cultures to test how well edits are made and how safe they are. The team will optimize delivery approaches such as virus-like particles and check for off-target effects and changes in gene activity. This preclinical work in human cells aims to build the technical foundation for possible future patient-directed preventive therapies.
Who could benefit from this research
Good fit: People who carry high-risk genetic variants at ARMS2/HTRA1 or CFH, or those with a strong family history of AMD, would be most relevant to this work.
Not a fit: Patients without the specific high-risk genotypes or those who already have advanced late-stage AMD with established vision loss are unlikely to benefit directly from these lab-based experiments.
Why it matters
Potential benefit: If successful, this could lead to a preventive approach that permanently lowers genetic risk for AMD before vision is lost.
How similar studies have performed: Similar gene-editing approaches have shown promise in cells and animal models, but applying base and prime editing to human eye tissues for AMD prevention is novel and still early-stage.
Where this research is happening
IOWA CITY, UNITED STATES
- UNIVERSITY OF IOWA — IOWA CITY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MULLINS, ROBERT FOSTER — UNIVERSITY OF IOWA
- Study coordinator: MULLINS, ROBERT FOSTER
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.