High-efficiency base editing for Stargardt disease in mice, non-human primates, and human retina tissue
A. Muller, J. Sullivan, W. Schwarzer, M. Wang, C. Park-Windhol, B. Klingler, J. Matsell, S. Hostettler, P. Galliker, M. Duman, Y. Hou, P. Balmer, T. Virag, L. A. Barrera, Q. Xu, , 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.
bioRxiv · Apr 17, 2023 · PreprintNeuroscience