Publications
Information about journal articles, updated at 17-11-2024, from platform CIÊNCIAVITAE.
Effective AAV-mediated gene replacement therapy in retinal organoids modelling AIPL1-associated LCA4
Sai, Hali; Ollington, Bethany; Rezek, Farah O.; Chai, Niuzheng; Lane, Amelia; Georgiadis, Tassos; Bainbridge, James; et al, Molecular Therapy - Nucleic Acids. 2024. http://dx.doi.org/10.1016/j.omtn.2024.102148 . 10.1016/j.omtn.2024.102148 . published Molecular Therapy - Nucleic Acids
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids
Afanasyeva, Tess A.V.; Athanasiou, Dimitra; Perdigao, Pedro R.L.; Whiting, Kae R.; Duijkers, Lonneke; Astuti, Galuh D.N.; Bennett, Jean; et al, Molecular Therapy - Methods & Clinical Development. 522 - 531. 29. 2023. http://dx.doi.org/10.1016/j.omtm.2023.05.012 . 10.1016/j.omtm.2023.05.012 . published Molecular Therapy - Methods & Clinical Development
A Novel Genetic Variant in MBD5 Associated with Severe Epilepsy and Intellectual Disability: Potential Implications on Neural Primary Cilia
Martins, Mariana; Oliveira, Ana Rafaela; Martins, Solange; Vieira, José Pedro; Perdigão, Pedro; Fernandes, Ana Rita; de Almeida, Luís Pereira; et al, International Journal of Molecular Sciences. 16. 24. 2023. http://dx.doi.org/10.3390/ijms241612603 . 10.3390/ijms241612603 . published International Journal of Molecular Sciences
Extracellular communication between brain cells through functional transfer of Cre mRNA
Rufino-Ramos, David; Leandro, K; Perdigão, Pedro (0F1E-EFD6-AF01); O'Brien, K; Pinto, MM; Santana, MM; van Solinge, TS; et al, Molecular Therapy. 2023. in press Molecular Therapy
Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease
Pedro Perdigão; Bethany Ollington; Hali Sai; Amy Leung; Almudena Sacristan-Reviriego; Jacqueline van der Spuy, International Journal of Molecular Sciences. 2023. https://www.mdpi.com/1422-0067/24/6/5912 . 10.3390/ijms24065912 . International Journal of Molecular Sciences
Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders
Benati, D; Leung, A; Perdigão, P; Toulis, V; van der Spuy, J; Recchia, A, International Journal of Molecular Sciences. 23. 23. 2023. https://www.mdpi.com/1422-0067/23/23/15276 . published International Journal of Molecular Sciences
Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis
Leung, Amy; Sacristan-Reviriego, Almudena; Perdigão, Pedro R.L.; Sai, Hali; Georgiou, Michalis; Kalitzeos, Angelos; Carr, Amanda-Jayne F.; et al, Stem Cell Reports. 2187 - 2202. 10. 17. 2022. http://dx.doi.org/10.1016/j.stemcr.2022.08.005 . 10.1016/j.stemcr.2022.08.005 . published Stem Cell Reports
Translational readthrough as a potential therapeutic for AIPL1-associated Leber Congenital Amaurosis in a patient-derived iPSC-retinal organoid model
Leung, A; Sacristan-Reviriego, A; Perdigão, Pedro (0F1E-EFD6-AF01); Sai, H; Georgiou, M; Kalitzeos, A; Carr, AJ; et al, bioRxiv. 2021. https://doi.org/10.1101/2021.12.17.473147 . published bioRxiv
CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs
Sladen, Paul E.; Perdigão, Pedro R.L.; Salsbury, Grace; Novoselova, Tatiana; van der Spuy, Jacqueline; Chapple, J. Paul; Yu-Wai-Man, Patrick; Cheetham, Michael E., Molecular Therapy - Nucleic Acids. 432 - 443. 26. 2021. http://dx.doi.org/10.1016/j.omtn.2021.08.015 . 10.1016/j.omtn.2021.08.015 . published Molecular Therapy - Nucleic Acids
Protein Delivery of Cell-Penetrating Zinc-Finger Activators Stimulates Latent HIV-1-Infected Cells
Perdigão, Pedro R.L.; Cunha-Santos, Catarina; Barbas, Carlos F.; Santa-Marta, Mariana; Goncalves, Joao, Molecular Therapy - Methods & Clinical Development. 145 - 158. 18. 2020. http://dx.doi.org/10.1016/j.omtm.2020.05.016 . 10.1016/j.omtm.2020.05.016 . published Molecular Therapy - Methods & Clinical Development
Inhibition of HIV replication through siRNA carried by CXCR4-targeted chimeric nanobody.
Cunha-Santos, C; Perdigao, PRL; Oliveira, JG; Cardoso, M; Manuel, A; Taveira, N; Goncalves, J, Cellular and molecular life sciences : CMLS. 2019. https://doi.org/10.1007/s00018-019-03334-8 . 10.1007/s00018-019-03334-8 . published Cellular and molecular life sciences : CMLS
Reactivation of Latent HIV-1 Expression by Engineered TALE Transcription Factors.
Perdigão, Pedro; Gaj, T; Santa-Marta, M; Barbas III, C; Goncalves, J, PloS one. 2016. http://europepmc.org/articles/PMC4774903 . 10.1371/journal.pone.0150037 . PloS one
5 12 out of 12 Publications
All projects
Information of exclusive responsibility of the researcher 17-11-2024 , from platform CIÊNCIAVITAE.
2024/01/01 - 2029/12/31
GeneT: The Gene Therapy CoE at the Center of Portugal
101059981
Researcher
Universidade de Coimbra
Funders: European Commission
2024/03/01 - 2025/02/28
Engineering RNA-Targeting CRISPR/Cas13 for the Treatment of Spinocerebellar Ataxia Type 3
25124
Principal investigator
Universidade de Coimbra
Funders: AFM-Téléthon
2023/08/01 - 2025/12/31
CiNTech - Technological Hub for Innovation, Translation and Industrialization of Complex Injectable Drugs
C644865576-00000005
Researcher
Universidade de Coimbra
Funders: European Commission
2022 - 2023
(Less)PlasticBrain: Bio Replacing Plasticizers - Impact in the Human Brain Development
(Less)PlasticBrain
Funders: Banco Santander Portugal
2022 - 2023
(Less)PlasticBrain: Bio Replacing Plasticizers - Impact in the Human Brain Development
(Less)PlasticBrain
Researcher
Universidade de Coimbra
Funders: Banco Santander Portugal
2022/01/01 - 2023/12/31
Genome editing to rescue Machado-Joseph disease by alternative splicing
EXPL/MED-NEU/0936/2021
Principal investigator
Universidade de Coimbra Centro de Neurociências e Biologia Celular
Funders: Fundação para a Ciência e a Tecnologia
2021/10/01 -
Machado-Joseph Disease iPSC Cell Models for Medium Throughput Drug Screening
CT 0091995
Post-doc
Universidade de Coimbra Centro de Neurociências e Biologia Celular
Funders: Institut Servier SAS
2020/06/01 - 2023/05/31
Rescue of inherited retinal degeneration by CRISPR base editing
5129 / 5130
Other
University College London Institute of Ophthalmology
Funders: Fight for Sight Foundation
2018/07/01 - 2021/09/30
Blocking neurodegeneration by spreading of gene silencers
PTDC/BTM-SAL/29716/2017
Universidade de Coimbra Centro de Neurociências e Biologia Celular
Funders: Fundação para a Ciência e a Tecnologia
2017/11/15 - 2019/07/12
Developing a photoreceptor model of AIPL1 LCA from patient derived induced pluripotent stem cells
R170015A
Post-doc
University College London Institute of Ophthalmology
Funders: Moorfields Eye Hospital
2016/01/01 - 2016/06/30
HIV-1 ERADICATION BY SITE-DIRECTED INTEGRATION
info:eu-repo/grantAgreement/FCT/3599-PPCDT/123638/PT
Research Fellow
Funders: Fundação para a Ciência e a Tecnologia
2014/03/01 - 2016/08/31
An innovative approach to deliver dual-targeting HIV entry inhibitors using cationic liposomes
HIVERA/0003/2013
Instituto de Medicina Molecular João Lobo Antunes; Instituto de Medicina Molecular João Lobo Antunes
Funders: Fundação para a Ciência e a Tecnologia
2014/03 - 2015/12
HIV-1 ERADICATION BY SITE-DIRECTED INTEGRATION
Provided by PTCRIS: 123638
Researcher
Funders: Fundação para a Ciência e a Tecnologia
2014/03 - 2016/02
An innovative approach to deliver dual-targeting HIV entry inhibitors using cationic liposomes
Provided by PTCRIS: 135858
Researcher
Funders: Fundação para a Ciência e a Tecnologia
2013/01 - 2015/03
Strategic Project - UI 775 - 2013-2014
Provided by PTCRIS: 133003
Funders: Fundação para a Ciência e a Tecnologia
2012/01/01 - 2012/12/31
Nanotechnology against viral latency: Sensor strategies to eliminate HIV-1 infected cells.
Design New Approaches to Cure HIV Infection (Round 6)
Universidade de Lisboa Instituto de Investigação do Medicamento
Funders: Bill & Melinda Gates Foundation
2012/01/01 - 2015/12
ERADICATION OF HIV-1 INFECTED CELLS BY TARGETING NANOPARTICLES CARRYING THERAPEUTIC PLASMIDS
info:eu-repo/grantAgreement/FCT/SFRH/SFRH2%FBD2%F819412%F2011/PT
PhD Student Fellow
Funders: Fundação para a Ciência e a Tecnologia
5 17 out of 17 projects
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