Nonviral gene editing for inherited retinal blindness in LCA16
Official title: The CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies Epqt Supplement
Funded at University of Wisconsin-Madison · NIH-11537957 · Data updated 2026-11-12
A patient-focused program uses a CRISPR-based, nonviral approach to fix a specific genetic cause of pediatric blindness in the retina.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Wisconsin-Madison NIH-funded |
| Lab location | 1 site (Madison, United States) |
| Project start | May 16, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $50,000 |
| NIH project number | 3U19NS132296-04S1 |
| Project ID | NIH-11537957 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young individuals with LCA16 caused by KCNJ13 mutations who are eligible for future gene therapy trials.
✕Less likely to help
Patients without LCA16 or without access to trial opportunities may not benefit from this specific approach.
What this research studies
This project aims to correct a mutation in the KCNJ13 gene that causes Leber congenital amaurosis type 16 (LCA16) using a refined CRISPR base editor delivered by silica nanocapsules directly to retinal cells. The work includes preclinical testing in mouse models and patient-derived retinal cells, with milestones toward scaled production and regulatory readiness. If successful, the approach could move toward trials in people with LCA16 to restore vision or stabilize retinal function.
Why it matters
Potential benefit: If successful, this could lead to a future therapy that preserves or improves vision for children with LCA16.
How similar studies have gone: CRISPR-based retinal therapies have shown promise in preclinical models, but this exact nonviral base-editing approach for LCA16 is in early stages.
Where this research is happening
-
University of Wisconsin-Madison
Madison, United States Active
Researchers
- Principal investigator: Saha, Krishanu — University of Wisconsin-Madison
- Study coordinator: Saha, Krishanu
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.