Cell BiologybioRxivPreprint
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…·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.
DOI 10.1101/2021.12.17.473147