We published our base editing paper on Bioarchives.
Stargardt disease, an inherited condition causing blindness, currently has no cure. Our team has developed a groundbreaking gene therapy using a dual AAV vector system to target the most common ABCA4 mutation, which leads to this disease. This new approach successfully corrected the mutation in human and in animal models. We achieved high levels of gene correction, with 37% in photoreceptors and 73% in retinal pigment epithelial cells. These results offer new hope for treating Stargardt disease and other neurodegenerative eye conditions in the future.
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