High-Efficiency Base Editing in the Retina of Primates and Human Tissues

High-precision Base Editing Technology in Primates and Human Retina Research Background Stargardt disease is a currently untreatable, inherited neurodegenerative disease that leads to macular degeneration and blindness due to loss-of-function mutations in the ABCA4 gene. The protein encoded by the ABCA4 gene is a membrane lipid flippase localized i...