NeurosciencebioRxivPreprint
High-efficiency base editing for Stargardt disease in mice, non-human primates, and human retina tissue
A. Muller, J. Sullivan, W. Schwarzer…·B. György
This study develops a base-editing strategy to correct the ABCA4 mutation in mice, non-human primates, and human retinal tissue, achieving high editing rates in vivo.
DOI 10.1101/2023.04.17.535579