Targeted viral gene tools for specific retinal cells
Novel strategies for developing AAV tools targeting specific cell types in mammalian retina
Researchers are building virus-based gene-delivery tools that switch on genes only in particular retinal cell types in adults to help study and someday treat retinal diseases.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-11080277 on NIH RePORTER |
What this research studies
The team is using adeno-associated viruses (AAVs) to deliver genetic labels or switches directly to single types of cells inside the retina. They design small DNA control elements (mini-promoters) based on genomic regulatory regions so the virus turns genes on only in the intended cell type, including rare cells like amacrine neurons. Because many candidate regulatory regions look promising but fail in practice, the researchers are testing new ways to find the best ones by looking at chromatin and transcription factor binding. Success would make it easier to map and manipulate specific retinal cells in lab and preclinical work, speeding development of targeted treatments.
Who could benefit from this research
Good fit: This work is most relevant to adults with retinal disorders or inherited retinal diseases who might benefit from future targeted gene-delivery treatments.
Not a fit: People with eye conditions unrelated to the retina or those with advanced, irreversible retinal scarring are unlikely to gain direct benefit from this project in the near term.
Why it matters
Potential benefit: If successful, this could enable much more precise targeting of retinal cells for gene therapies and improve understanding of many retinal diseases.
How similar studies have performed: AAV-based gene therapies have succeeded for some retinal conditions (for example RPE65-related vision loss), but reliably targeting rare retinal subtypes with cell-type-specific control sequences remains experimental.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Wang, Sui — Stanford University
- Study coordinator: Wang, Sui
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.