Translational readthrough as a potential therapeutic for AIPL1-associated Leber Congenital Amaurosis in a patient-derived iPSC-retinal organoid model
A. Leung, A. Sacristan-Reviriego, P. Perdigao, H. Sai, M. Georgiou, A. Kalitzeos, A.-J. F. Carr, P. J. Coffey, M. Michaelides, J. Bainbridge, M. E. Cheetham, J. van der Spuy
This study uses patient-derived iPSC-retinal organoids to investigate translational readthrough as a therapy for LCA4 caused by AIPL1 mutations.
bioRxiv · Dec 17, 2021 · PreprintCell Biology