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2011 2
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Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis.
Leung A, Sacristan-Reviriego A, Perdigão PRL, Sai H, Georgiou M, Kalitzeos A, Carr AF, Coffey PJ, Michaelides M, Bainbridge J, Cheetham ME, van der Spuy J. Leung A, et al. Among authors: sacristan reviriego a. Stem Cell Reports. 2022 Oct 11;17(10):2187-2202. doi: 10.1016/j.stemcr.2022.08.005. Epub 2022 Sep 8. Stem Cell Reports. 2022. PMID: 36084639 Free PMC article.
Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4.
Sai H, Ollington B, Rezek FO, Chai N, Lane A, Georgiadis T, Bainbridge J, Michaelides M, Sacristan-Reviriego A, Perdigão PRL, Leung A, van der Spuy J. Sai H, et al. Among authors: sacristan reviriego a. Mol Ther Nucleic Acids. 2024 Feb 13;35(1):102148. doi: 10.1016/j.omtn.2024.102148. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38439910 Free PMC article.
15 results