Hali Sai

发表

Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis

M. Michaelides, M. Cheetham, J. Bainbridge, 2022, Stem cell reports.

Congenital Amaurosis in a patient-derived iPSC-retinal organoid model Author List

P. Coffey, M. Michaelides, M. Cheetham, 2021 .

Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease

Almudena Sacristán-Reviriego, J. van der Spuy, A. Leung, 2023, International journal of molecular sciences.

Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4

Almudena Sacristán-Reviriego, J. van der Spuy, P. Perdigão, 2024, Molecular therapy. Nucleic acids.