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CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids.
Afanasyeva TAV, Athanasiou D, Perdigao PRL, Whiting KR, Duijkers L, Astuti GDN, Bennett J, Garanto A, van der Spuy J, Roepman R, Cheetham ME, Collin RWJ. Afanasyeva TAV, et al. Among authors: perdigao prl. Mol Ther Methods Clin Dev. 2023 May 17;29:522-531. doi: 10.1016/j.omtm.2023.05.012. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37305852 Free PMC article.
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: perdigao prl. 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.
Extracellular communication between brain cells through functional transfer of Cre mRNA.
Rufino-Ramos D, Leandro K, Perdigão PRL, O'Brien K, Pinto MM, Santana MM, van Solinge TS, Mahjoum S, Breakefield XO, Breyne K, de Almeida LP. Rufino-Ramos D, et al. Among authors: perdigao prl. bioRxiv [Preprint]. 2023 Jan 31:2023.01.29.525937. doi: 10.1101/2023.01.29.525937. bioRxiv. 2023. PMID: 36811091 Free PMC article. Updated. Preprint.
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: perdigao prl. 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.