Publications by authors named "Pedro Perdigao"

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Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids.
Amy Leung, Pedro R L Perdigão, Almudena Sacristan-Reviriego, Paul E Sladen, Erika A Aguzzi

Mol Ther Nucleic Acids· December 2025

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Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads.
Kevin Leandro, David Rufino-Ramos, Koen Breyne, Emilio Di Ianni, Sara M Lopes

Adv Drug Deliv Rev· August 2024

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Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4.
Hali Sai, Bethany Ollington, Farah O Rezek, Niuzheng Chai, Amelia Lane

Mol Ther Nucleic Acids· March 2024

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A Novel Genetic Variant in Associated with Severe Epilepsy and Intellectual Disability: Potential Implications on Neural Primary Cilia.
Mariana Martins, Ana Rafaela Oliveira, Solange Martins, José Pedro Vieira, Pedro Perdigão

Int J Mol Sci· August 2023

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Extracellular communication between brain cells through functional transfer of Cre mRNA mediated by extracellular vesicles.
David Rufino-Ramos, Kevin Leandro, Pedro R L Perdigão, Killian O'Brien, Maria Manuel Pinto

Mol Ther· July 2023

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CRISPR-Cas9 correction of a nonsense mutation in rescues lebercilin expression and localization in human retinal organoids.
Tess A V Afanasyeva, Dimitra Athanasiou, Pedro R L Perdigao, Kae R Whiting, Lonneke Duijkers

Mol Ther Methods Clin Dev· June 2023

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Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease.
Pedro R L Perdigão, Bethany Ollington, Hali Sai, Amy Leung, Almudena Sacristan-Reviriego

Int J Mol Sci· March 2023

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Extracellular communication between brain cells through functional transfer of Cre mRNA.
David Rufino-Ramos, Kevin Leandro, Pedro R L Perdigão, Killian O'Brien, Maria Manuel Pinto

bioRxiv· January 2023

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Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders.
Daniela Benati, Amy Leung, Pedro Perdigao, Vasileios Toulis, Jacqueline van der Spuy

Int J Mol Sci· December 2022

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Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis.
Amy Leung, Almudena Sacristan-Reviriego, Pedro R L Perdigão, Hali Sai, Michalis Georgiou

Stem Cell Reports· October 2022

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CRISPR-Cas9 correction of c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs.
Paul E Sladen, Pedro R L Perdigão, Grace Salsbury, Tatiana Novoselova, Jacqueline van der Spuy

Mol Ther Nucleic Acids· December 2021

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Protein Delivery of Cell-Penetrating Zinc-Finger Activators Stimulates Latent HIV-1-Infected Cells.
Pedro R L Perdigão, Catarina Cunha-Santos, Carlos F Barbas, Mariana Santa-Marta, Joao Goncalves

Mol Ther Methods Clin Dev· September 2020

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Inhibition of HIV replication through siRNA carried by CXCR4-targeted chimeric nanobody.
Catarina Cunha-Santos, Pedro Ricardo Lucas Perdigao, Francisco Martin, Joana Gomes Oliveira, Miguel Cardoso

Cell Mol Life Sci· July 2020

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Gene and Cell Therapy for AIPL1-Associated Leber Congenital Amaurosis: Challenges and Prospects.
Pedro R L Perdigao, Jacqueline van der Spuy

Adv Exp Med Biol· February 2020

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Reactivation of Latent HIV-1 Expression by Engineered TALE Transcription Factors.
Pedro Perdigão, Thomas Gaj, Mariana Santa-Marta, Carlos F Barbas, Joao Goncalves

PLoS One· July 2016

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