| CRISPR-Cas9 correction of OPA1 c. 1334G> A: p. R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs PE Sladen, PRL Perdigão, G Salsbury, T Novoselova, J van der Spuy, ... Molecular Therapy-Nucleic Acids 26, 432-443, 2021 | 23 | 2021 |
| Inhibition of HIV replication through siRNA carried by CXCR4-targeted chimeric nanobody C Cunha-Santos, PRL Perdigao, F Martin, JG Oliveira, M Cardoso, ... Cellular and Molecular Life Sciences 77 (14), 2859-2870, 2020 | 16 | 2020 |
| Reactivation of latent HIV-1 expression by engineered TALE transcription factors P Perdigão, T Gaj, M Santa-Marta, CF Barbas III, J Goncalves PloS one 11 (3), e0150037, 2016 | 16 | 2016 |
| Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis A Leung, A Sacristan-Reviriego, PRL Perdigão, H Sai, M Georgiou, ... Stem Cell Reports 17 (10), 2187-2202, 2022 | 15 | 2022 |
| CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids TAV Afanasyeva, D Athanasiou, PRL Perdigao, KR Whiting, L Duijkers, ... Molecular Therapy-Methods & Clinical Development 29, 522-531, 2023 | 7 | 2023 |
| Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease PRL Perdigão, B Ollington, H Sai, A Leung, A Sacristan-Reviriego, ... International Journal of Molecular Sciences 24 (6), 5912, 2023 | 6 | 2023 |
| Protein Delivery of Cell-Penetrating Zinc-Finger Activators Stimulates Latent HIV-1-Infected Cells PRL Perdigão, C Cunha-Santos, CF Barbas III, M Santa-Marta, ... Molecular Therapy-Methods & Clinical Development 18, 145-158, 2020 | 5 | 2020 |
| Gene and Cell Therapy for AIPL1-Associated Leber Congenital Amaurosis: Challenges and Prospects PRL Perdigao, J van der Spuy Retinal Degenerative Diseases, 97-101, 2019 | 4 | 2019 |
| Extracellular communication between brain cells through functional transfer of Cre mRNA mediated by extracellular vesicles D Rufino-Ramos, K Leandro, PRL Perdigão, K O'Brien, MM Pinto, ... Molecular Therapy, 2023 | 3 | 2023 |
| Induced pluripotent stem cells and genome-editing tools in determining gene function and therapy for inherited retinal disorders D Benati, A Leung, P Perdigao, V Toulis, J van der Spuy, A Recchia International Journal of Molecular Sciences 23 (23), 15276, 2022 | 3 | 2022 |
| Extracellular communication between brain cells through functional transfer of Cre mRNA D Rufino-Ramos, K Leandro, PRL Perdigão, K O’Brien, MM Pinto, ... Biorxiv, 2023 | 1 | 2023 |
| A Novel Genetic Variant in MBD5 Associated with Severe Epilepsy and Intellectual Disability: Potential Implications on Neural Primary Cilia M Martins, AR Oliveira, S Martins, JP Vieira, P Perdigão, AR Fernandes, ... International Journal of Molecular Sciences 24 (16), 12603, 2023 | | 2023 |
| Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease K Leandro, D Rufino-Ramos, T D'Amelio, A Margarida Salgueiro, S Lopes, ... HUMAN GENE THERAPY 33 (23-24), A179-A179, 2022 | | 2022 |
| Modelling Machado-Joseph Disease in patientderived isogenic lines by CRISPR/Cas9-mediated correction of ATXN3-CAG expansion P Perdigao, C Matos, S Lopes, R Fernandes, M Lopes, A Conceicao, ... HUMAN GENE THERAPY 33 (23-24), A119-A119, 2022 | | 2022 |
| Insomnia patient-derived iPSC neurons with potential for disease modeling C Carvalhas-Almeida, LP de Almeida, C Cavadas, P Perdigão, AR Álvaro Sleep Medicine 100, S124-S125, 2022 | | 2022 |
| 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, PRL Perdigão, H Sai, M Georgiou, ... bioRxiv, 2021.12. 17.473147, 2021 | | 2021 |
| Investigation of Ataluren (PTC124) as a potential therapeutic for LCA4 in a patient-derived iPS-retinal organoid model A Leung, A Sacristan-Reviriego, P Perdigao, JWB Bainbridge, ... Investigative Ophthalmology & Visual Science 62 (8), 1671-1671, 2021 | | 2021 |
| Gene-targeted strategies to eliminate HIV latent cells through synthetic activators and suicide lentivectors PRL Perdigão PQDT-Global, 2017 | | 2017 |
| Specific elimination of HIV-1 infected cells using Tat/Rev-dependent circuit PRL Perdigão | | 2011 |
