Efficient gene editing in the retina in primates and human tissues
- rani mizrahi
- Oct 29
- 1 min read
Published in Nature Medicine
A new study presents a breakthrough in the treatment of Stargate disease – a hereditary degenerative disease of the retina, which causes blindness due to a mutation in the ABCA4 gene. The researchers developed a dual AAV virus-based system, which allows for precise genetic base editing to correct the most common mutation in the disease. The tool was tested in advanced models of human retinal cells, organoids, as well as in primates, and achieved extremely high repair rates – up to 87% in retinal pigment epithelial cells. These findings indicate real potential for clinical translation, not only for Stargate disease but also for other eye diseases that can be treated through advanced gene editing.
