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Identification

Personal identification

Full name
Pedro Ricardo Lucas Perdigão

Citation names

  • Perdigão, PRL
  • Perdigão, P
  • Pedro Perdigão

Author identifiers

Ciência ID
0F1E-EFD6-AF01
ORCID iD
0000-0003-2943-3515
Education
Degree Classification
2020/11/09 - 2020/11/20
Concluded
 Modular Training in Laboratory Animal Science (Curso médio)
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2012/01/01 - 2017/06/20
Concluded
 Farmácia (Doutoramento)
Universidade de Lisboa Faculdade de Farmácia, Portugal
"Gene-targeted strategies to eliminate HIV latent cells through synthetic activators and suicide lentivectors" (THESIS/DISSERTATION)
 Aprovado com distinção e louvor
2009/09/20 - 2011/11/21
Concluded
 Biologia Molecular e Genética (Mestrado)
Universidade de Lisboa Faculdade de Ciências, Portugal
"Specific elimination of HIV-1 infected cells using a Tat/Rev-dependent circuit" (THESIS/DISSERTATION)
 17 (0-20)
2005/09/20 - 2009/07/09
Concluded
 Bioquímica (Licenciatura)
Universidade de Lisboa Faculdade de Ciências, Portugal
 15 (0-20)
Affiliation

Science

Category
Host institution		 Employer
2023/08/01 - Current Auxiliary Researcher (Research) Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2019/07/15 - 2023/07/31 Postdoc (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2017/11/15 - 2019/07/12 Postdoc (Research) University College London Institute of Ophthalmology, United Kingdom
University College London Institute of Ophthalmology, United Kingdom
2010/08/01 - 2017/06/20 Researcher (Research) Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
Universidade de Lisboa Faculdade de Farmácia, Portugal
2013/11/07 - 2014/10/17 Visiting Researcher (Research) Scripps Research Institute, United States
Scripps Research Institute, United States

Others

Category
Host institution		 Employer
2020/03 - 2020/06 Research volunteer for COVID-19 task force Universidade de Coimbra, Portugal
Projects

Grant

Designation Funders
2024/01/01 - Current GeneT: The Gene Therapy CoE at the Center of Portugal
101059981
Researcher
Universidade de Coimbra, Portugal
 European Commission
Ongoing
2026/01/01 - 2028/12/31 Potassium channel Kv7.2 complexes as therapeutic targets in epilepsy
2023.18102.ICDT
Researcher
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
 Fundação para a Ciência e a Tecnologia
Ongoing
2025/07/31 - 2028/07/28 (Re)modeling of (Strategies) for (C)ircadian Rhythm and (G)ene Therapy in Fatal Familial Insomnia
COMPETE2030-FEDER-00698300
Researcher
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
 Fundação para a Ciência e a Tecnologia
Ongoing
2025 - 2028 REACT-FFI: (Re)shaping (A)pproaches for (C)ircadian Rhythm and gene (T)herapy in Familial Fatal Insomnia
15868 (MPr-2023-12)
Universidade de Coimbra, Portugal

Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
 Fundação para a Ciência e a Tecnologia
2026/04/01 - 2027/03/31 Non-Cell Autonomous Effects in SCA3: Elucidating Astrocyte-Induced Neuronal Dysfunction in a Human Patient-Derived Model
1564453
Principal investigator
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
 National Ataxia Foundation
Ongoing
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, Portugal
 AFM-Téléthon
2023/03/01 - 2024/08/31 Pre-clinical evaluation of the brain-targeting AAV-PHP.eB encoding cholesterol hydroxylase CYP46A1 as a therapeutic strategy for spinocerebellar ataxia type 3 – towards the understanding of the neuroprotective mechanism
2022.04788.PTDC
Universidade de Coimbra, Portugal
 Fundação para a Ciência e a Tecnologia
Ongoing
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, Portugal
 Fundação para a Ciência e a Tecnologia
Concluded
2020/06/01 - 2023/05/31 Rescue of inherited retinal degeneration by CRISPR base editing
5129 / 5130
Co-Applicant
University College London Institute of Ophthalmology, United Kingdom
 Fight for Sight Foundation
Ongoing
2022 - 2023 (Less)PlasticBrain: Bio Replacing Plasticizers - Impact in the Human Brain Development
(Less)PlasticBrain
Researcher
Universidade de Coimbra, Portugal
 Banco Santander Portugal
Concluded
2022 - 2023 (Less)PlasticBrain: Bio Replacing Plasticizers - Impact in the Human Brain Development
(Less)PlasticBrain
 Banco Santander Portugal
2016/01/01 - 2016/06/30 HIV-1 ERADICATION BY SITE-DIRECTED INTEGRATION
info:eu-repo/grantAgreement/FCT/3599-PPCDT/123638/PT
Research Fellow
Fundação para a Ciência e a Tecnologia
Concluded
2014/03 - 2016/02 An innovative approach to deliver dual-targeting HIV entry inhibitors using cationic liposomes
Provided by PTCRIS: 135858
Researcher
Fundação para a Ciência e a Tecnologia
2014/03 - 2015/12 HIV-1 ERADICATION BY SITE-DIRECTED INTEGRATION
Provided by PTCRIS: 123638
Researcher
Fundação para a Ciência e a Tecnologia
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
Fundação para a Ciência e a Tecnologia
Concluded
2013/01 - 2015/03 Strategic Project - UI 775 - 2013-2014
Provided by PTCRIS: 133003
 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, Portugal
 Bill & Melinda Gates Foundation
Concluded

Contract

Designation Funders
2023/08/01 - Current CiNTech - Technological Hub for Innovation, Translation and Industrialization of Complex Injectable Drugs
C644865576-00000005
Researcher
Universidade de Coimbra, Portugal
 European Commission
Ongoing
2021/01/01 - 2025/12/31 Centro de Inovação em Biomedicina e Biotecnologia - Laboratório Associado
LA/P/0058/2020
10.54499/LA/P/0058/2020
Universidade de Coimbra, Portugal

Universidade Nova de Lisboa Laboratório de Instrumentação Engenharia Biomédica e Física da Radiação, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Fundação para a Ciência e a Tecnologia
Ongoing
2020/01/01 - 2024/12/31 Centro de Inovação em Biomedicina e Biotecnologia
UIDP/04539/2020
10.54499/UIDP/04539/2020
Universidade de Coimbra, Portugal

Universidade de Coimbra Instituto de Investigação Clínica e Biomédica de Coimbra, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Fundação para a Ciência e a Tecnologia
Concluded
2021/10/01 - 2023/07/31 Machado-Joseph Disease iPSC Cell Models for Medium Throughput Drug Screening
CT 0091995
Post-doc
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Institut Servier SAS
Ongoing
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, Portugal
 Fundação para a Ciência e a Tecnologia
Ongoing
2019/01/01 - 2019/12/31 Instituto de Investigação do Medicamento
UID/DTP/04138/2019
154706UID
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal

Universidade de Lisboa Faculdade de Farmácia, Portugal
 Fundação para a Ciência e a Tecnologia
Concluded
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, United Kingdom
 Moorfields Eye Hospital
Concluded
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, Portugal

Instituto de Medicina Molecular João Lobo Antunes, Portugal
 Fundação para a Ciência e a Tecnologia
Concluded
Outputs

Publications

Book chapter
  1. Perdigão, Pedro; van der Spuy, J. "Gene and Cell Therapy for AIPL1-Associated Leber Congenital Amaurosis: Challenges and Prospects". 2019.
    10.1007/978-3-030-27378-1_16
Conference abstract
  1. Diana Lobo; Luís Quintino; Pinto, Marcos; Rita Almeida; Ana Carolina Silva; Carina Henriques; Rodrigo N. Ribeiro; et al. "CRISPR activation as a new gene therapy strategy for Machado-Joseph disease". Paper presented in BEB Day 2025, Coimbra, 2025.
    Published
  2. Perdigão, Pedro (0F1E-EFD6-AF01); Gaj, Thomas; Barbas III, Carlos F; Santa-Marta, Mariana; Gonçalves, João. "Reactivation of latent HIV-1 expression by engineered TALE transcription factors". Paper presented in European Society of Gene and Cell Therapy and Finland Society of Gene and Cell Therapy Collaborative Congress, Helsinki, 2015.
    Published
Conference poster
  1. Patrício, D; Perdigão, PRL; Sousa, Inês S; Peter M.J. Quinn; Ambrósio, A.F.; Marques, João Pedro; Alves, C. Henrique. "Modelling EYS-associated Retinitis Pigmentosa with patient-derived photoreceptor precursor cells and retinal organoids". Paper presented in Retina4Future 1st Scientific Meeting (COST Action 24105), 2026.
  2. Pereira, Ana Maria; Pires, Mariana; Tomás, Inês; Silva, Ana Carolina; Almeida, Rita; Fernandes, Ana Rita; Pena, Frederico; et al. Corresponding author: Perdigão, PRL. "ENGINEERING RNA-TARGETING CRISPR/CAS13 FOR ALLELE-SPECIFIC ATXN3 KNOCKDOWN FOR TREATMENT OF SPINOCEREBELLAR ATAXIA TYPE 3". Paper presented in 29º Reunião Anual da Sociedade Portuguesa de Genética Humana, Coimbra, Portugal, 2025.
  3. RODRIGUES, M. T.; Paulo, Cristiana; Valdeira, A. S. C.; Santos Silva, Francisca; Marques, Bruno; Simões-Henriques, Carla; Gaspar, Laetitia S; et al. "Comparative Evaluation of mRNA Delivery Strategies in HEK293T Cells and Human Dermal Fibroblasts Using Linear, Self-Amplifying, and Circular RNA". Paper presented in 13th International mRNA Health Conference, 2025.
  4. RODRIGUES, M. T.; Paulo, Cristiana; Valdeira, A. S. C.; Santos Silva, Francisca; Marques, Bruno; Simões-Henriques, Carla; Gaspar, Laetitia S; et al. "Assessment of mRNA delivery approaches in HEK293T cells and Human Dermal Fibroblasts (HDF) with linear mRNA, self-amplifying mRNA (saRNA), and circular RNA (cRNA)". Paper presented in 29th Annual Meeting of the Portuguese Society of Human Genetics, 2025.
  5. Marques, Bruno; Simões-Henriques, Carla; RODRIGUES, M. T.; Paulo, Cristiana; Perdigão, PRL; Sónia P. Duarte; Miranda, CO; et al. "Optimizing Self-Amplifying RNA Production: A Comparative Study of IVT Methods, Rational Sequence Design, and Nucleotide Modifications". Paper presented in 13th International mRNA Health Conference, 2025.
  6. Simões-Henriques, Carla; Marques, Bruno; Paulo, Cristiana; Pereira, Dina; RODRIGUES, M. T.; Rita Perfeito; Sónia P. Duarte; et al. "Cost-Effective Optimization of Linear mRNA Synthesis: IVT Conditions, Sequence Engineering, and Nucleotide Modifications". Paper presented in 13th International mRNA Health Conference, 2025.
  7. Simões-Henriques, Carla; Marques, Bruno; Paulo, Cristiana; Pereira, Dina; RODRIGUES, M. T.; Rita Perfeito; Sónia P. Duarte; et al. "Cost-effective linear mRNA production through optimized in vitro transcription, rational sequence engineering, and chemically modified nucleotides". Paper presented in 29th Annual Meeting of the Portuguese Society of Human Genetics, 2025.
  8. Marques, Bruno; Simões-Henriques, Carla; RODRIGUES, M. T.; Paulo, Cristiana; Perdigão, PRL; Sónia P. Duarte; Miranda, CO; et al. "Optimized cost-effective production of self-amplifying RNA for RNA therapeutics: in vitro transcription conditions, sequence engineering, and chemically modified nucleotides". Paper presented in 29th Annual Meeting of the Portuguese Society of Human Genetics, 2025.
  9. Santos Silva, Francisca; Valdeira, A. S. C.; Paulo, Cristiana; RODRIGUES, M. T.; Gaspar, Laetitia S; Miranda, CO; Rita Perfeito; et al. "Advancing Circular RNA Therapeutics via Cost-Effective Production, Rational Sequencing Design, and Nucleotide Modifications". Paper presented in 13th International mRNA Health Conference, 2025.
  10. Valdeira, A. S. C.; Santos Silva, Francisca; Paulo, Cristiana; RODRIGUES, M. T.; Gaspar, Laetitia S; Miranda, CO; Rita Perfeito; et al. "Rational design and modified nucleotides enable cost-effective production of therapeutic circular RNA". Paper presented in 29th Annual Meeting of the Portuguese Society of Human Genetics, 2025.
  11. Henriques, Daniel; Moreira, Ricardo; Pena, Frederico; Perdigão, PRL; Kirkeby, Agnete; Nóbrega, Clévio David Rodrigues ; Pereira de Almeida, Luis ; Mendonça, Liliana. "Strategies to enhance stem cell migration and differentiation upon cerebellar transplantation". Paper presented in 35th Annual Symposium of the Network for European CNS Transplantation and Restoration - NECTAR 2025, 2025.
  12. Pereira, A.; Pires, M.; Silva, Ana Carolina; Almeida, Rita B.; Fernandes, Ana Rita; Pena, Frederico; Tomás, I.; et al. "Engineering RNA-targeting CRISPR/Cas13 ribonucleases for allele-specific knockdown of mutant ATXN3 for treatment of Spinocerebellar Ataxia Type 3". Paper presented in ESGCT/SETGYC Collaborative Congress, 2025.
  13. Pena, Frederico; Fernandes, Ana Rita; Miguel M. Lopes; Núñez, Jisette; Faro, Rosario; Pires, Mariana; Pereira, Ana Maria; et al. Corresponding author: Perdigão, PRL. "Establishment of a scalable neuronal reporter model of Machado-Joseph disease based on gene-edited patient cells as a platform for high-throughput drug repurposing". Paper presented in European Society of Gene and Cell Therapy and Finland Society of Gene and Cell Therapy Collaborative Congress, 2025.
  14. Henriques, Daniel; Moreira, Ricardo; Pena, Frederico; Pedro Perdigão; Agnete Kirkeby; Luís Pereira de Almeida; Liliana Mendonça. "Generating Cerebellar Neuronal Progenitors for cerebellar transplantation in spinocerebellar ataxia type 3 (SCA3)". Paper presented in International Congress for Ataxia Research, 2024.
  15. Gaspar, Laetitia S; Marques, Bruno; De Luca, Pasqualino; Pereira, Dina; Perdigão, PRL; Lopes, Sara M. ; Rita Perfeito; et al. "Cost-Effective Optimization of Self-Amplifying mRNA Production: Comparative Analysis of Commercial Kits and In Vitro Transcription Conditions". Paper presented in 12th mRNA Health Conference, 2024.
  16. Pereira, Dina; Valdeira, A. S. C.; De Luca, Pasqualino; Gaspar, Laetitia S; Lopes, Sara M. ; Perdigão, PRL; Santos Silva, Francisca; et al. "Enhancing circular mRNA yield through in vitro transcription optimization". 2024.
  17. De Luca, Pasqualino; Perdigão, PRL; Gaspar, Laetitia S; Pereira, Dina; Marques, Bruno; Lopes, Sara M. ; Melo, Alexandra A.; et al. "Maximizing mRNA vaccine production by optimizing in vitro transcription processes". Paper presented in 12th mRNA Health Conference, 2024.
  18. Sónia P. Duarte; Rita Perfeito; Miranda, CO; RODRIGUES, M. T.; Paulo, Cristiana; Pereira, Dina; De Luca, Pasqualino; et al. "Functional assays to optimize transfection efficiency for linear, circular and self-amplifying mRNA". Paper presented in 12th mRNA Health Conference, 2024.
  19. Catarina Oliveira Miranda; Perfeito, Rita; Sónia P. Duarte; Paulo, Cristiana; RODRIGUES, MARIANA TEIXEIRA; Gaspar, Laetitia S; Pereira, Dina; et al. "Comparative analysis of transfection kits for linear, self-amplifying, and circular RNA". Paper presented in ESGCT 31st Annual Congress, 2024.
  20. Gaspar, Laetitia S; Marques, Bruno; Pereira, Dina; De Luca, Pasqualino; Pedro Perdigão; Lopes, Sara; Perfeito, Rita; et al. "Maximizing Yield and Functionality in saRNA Production: A Comparative Study of Commercial Kits and In Vitro Transcription Conditions". Paper presented in ESGCT 31st Annual Congress, 2024.
  21. De Luca, Pasqualino; Lopes, Sara; Pereira, Dina; Gaspar, Laetitia S; Marques, Bruno; Pedro Perdigão; Melo, Alexandra A.; et al. "Optimizing in vitro transcription methods for enhanced mRNA-based therapeutics". Paper presented in ESGCT 31st Annual Congress, 2024.
  22. Pereira, Dina; Valdeira, A. S. C.; Santos Silva, Francisca; Gaspar, Laetitia S; De Luca, Pasqualino; Pedro Perdigão; Lopes, Sara; et al. "A comparative analysis of commercial kits for enhanced circular mRNA production: yield, profile, and in vitro assessment". Paper presented in ESGCT 31st Annual Congress, 2024.
  23. Leandro, Kevin; Rufino-Ramos, David; Lopes, Sara M.; Santos, Paulo; Silva, Ana Carolina; Fernandes, Ana Rita; Henriques, Carina; et al. Corresponding author: Pereira de Almeida, Luis. "Engineered extracellular vesicles for delivery of CRISPR-Cas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3.". Paper presented in Extracellular Vesicles Gordon Research Conference 2024 - Structure and Function, Technology Development, and Clinical Application, Newry, Maine, USA, 2024.
  24. Leandro, Kevin; Rufino-Ramos, David; Lopes, Sara M. ; Santos, Paulo; Silva, Ana Carolina; Fernandes, Ana Rita; Henriques, Carina; et al. Corresponding author: Pereira de Almeida, Luis. "Engineered extracellular vesicles for delivery of CRISPR-Cas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3. Extracellular Vesicles Gordon Research Seminar - Structure and Function, Technology Development, and Clinical Application". Paper presented in Extracellular Vesicles Gordon Research Conference 2024 - Structure and Function, Technology Development, and Clinical Application, 2024.
  25. Fernandes, Ana Rita; Diana Adão; Lopes, Sara M. ; Pena, Frederico; De Luca, Pasqualino; Henriques, Daniel; Mendonça, Liliana; et al. "Engineering of patient-derived isogenic neuronal models by CRISPR/Cas9 to unravel the impact of ATXN3 knockout in Spinocerebellar Ataxia Type 3". Paper presented in International Congress for Ataxia Research 2024, 2023.
  26. Lobo, Diana Duarte; Luís Quintino; Ana Carolina Silva; Carina Henriques; Ribeiro, Rodrigo; Lopes, Sara M. ; Pedro Perdigão; et al. Corresponding author: Pereira de Almeida, Luis Fernando Morgado. "CRISPR activation as a therapeutic tool for Spinocerebellar ataxia type 3 (SCA3)". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  27. Leandro, Kevin; Rufino-Ramos, David; Lopes, Sara M. ; Pasqua, C; Santos, P; Silva, Ana Carolina; Lobo, Diana Duarte; et al. Corresponding author: Pereira de Almeida, Luis. "Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease/Spinocerebellar Ataxia Type 3". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  28. Fernandes, Ana Rita; Adão, Diana; Lopes, Sara M. ; Henriques, Daniel; Pena, Frederico; Dias, Miguel; Magda M Santana; Pedro Perdigão; Pereira de Almeida, Luis . Corresponding author: Pereira de Almeida, Luis. "Engineering isogenic neuronal models of ATXN3 knockout in Machado-Joseph Disease patient cells by CRISPR/Cas9". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  29. De Luca, Pasqualino; Diana Adão; Pedro Perdigão; Pereira de Almeida, Luis ; Magda M Santana. "Neuronal maturation of induced pluripotent stem cell is altered in Machado Joseph-Disease cell lines". Paper presented in ETRS & SPCE-TC joint meeting 2023, 2023.
  30. Henriques, Daniel; Ricardo Moreira; Pedro Perdigão; Agnete Kirkeby; Pereira de Almeida, L; Liliana S. Mendonça. "Induced pluripotent stem cell-derived Cerebellar Neuronal Progenitors for transplantation in Machado-Joseph Disease". Paper presented in FENS Regional meeting 2023, 2023.
  31. Perdigão, PRL; Dias, Miguel; Lopes, Sara M. ; Fernandes, Ana Rita; Pena, Frederico; Miguel M. Lopes; Nobre, Rui Jorge; Gaj, Thomas; Pereira de Almeida, Luis. "Engineering CRISPR-Based Editors for Treatment of Machado-Joseph Disease". Paper presented in ASGCT 26th Annual Meeting, 2023.
  32. Carvalhas-Almeida, Catarina; Ferreira, Mafalda; Moita, Joaquim; Serra, Joana; Pereira de Almeida, Luis ; Cavadas, Claudia; Pedro Perdigão; Álvaro, Ana Rita. "Chronic insomnia: generation of iPSCs-derived neuronal disease model and characterization of peripheral circadian rhythms of patients". Paper presented in eSleep, 2023.
  33. Perdigão, PRL; Matos, Carlos A; Lopes, Sara M. ; Fernandes, Ana Rita; Miguel M. Lopes; Conceição, A; Faro, R; et al. "Modelling Machado-Joseph Disease in patient-derived isogenic lines by CRISPR/Cas9 homology directed repair.". Paper presented in 26th Annual Portuguese Society of Human Genetics Meeting, 2022.
  34. Martins, Mariana; Oliveira, Ana Rafaela; Cammarata, Giuseppe; Martins, Solange; Pedro Perdigão; Guedes, Joana; Cardoso, Ana Luísa; et al. "BrainCils: Exploring the missing link between neuronal primary cilia dysfunction and neurodevelopmental disorders: hints from dental stem cell-derived brain organoids". Paper presented in Synapse Meeting, 2022.
  35. Perdigão, P; Matos, Carlos A; Lopes, Sara; Fernandes, Ana Rita; Lopes, Miguel; Conceição, André; Faro, Rosario; et al. "Modelling Machado-Joseph Disease in patient-derived isogenic lines by CRISPR/Cas9-mediated correction of ATXN3-CAG expansion". Paper presented in ESGCT 29th Annual Congress, European Society of Gene and Cell Therapy, Edinburgh, 2022, 2022.
  36. Henriques, Daniel; Moreira, Ricardo; Pedro Perdigão; Agnete Kirkeby; Pereira de Almeida, Luis ; Mendonça, Liliana S. "Generation and characterization of cerebellar neuronal progenitors for Machado-Joseph Disease treatment". Paper presented in The 29th Annual Meeting of the European Society of Gene & Cell Therapy – ESGCT 2022, 2022.
  37. Leandro, Kevin; Rufino-Ramos, David; D'Amelio, T.; Salgueiro, Margarida A.; Lopes, Sara M. ; Pereira, Dina; Martins, M. I.; et al. Corresponding author: Pereira de Almeida, Luis. "Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease". Paper presented in 29th Congress of the European Society for Gene and Cell Therapy (ESGCT) in collaboration with BSGCT; Edinburgh, Scotland, United Kingdom, 2022.
  38. Carvalhas-Almeida, Catarina; Pereira de Almeida, Luis ; Cavadas, Claudia; Pedro Perdigão; Álvaro, Ana Rita. "Characterization iPSC-derived neuronal cells with potential for disease modeling of human insomnia". Paper presented in 12th International Meeting of the Portuguese Society for Stem Cells and Cell Therapies, 2022.
  39. Carvalhas de Almeida, Catarina; Pereira de Almeida, Luis ; Cavadas, Claudia; Perdigão, Pedro*; Álvaro, Ana Rita* . Corresponding author: Perdigão, Pedro*. "Insomnia patient-derived iPSC neurons with potential for disease modeling". Paper presented in World Spleep Congress 2022, Rome, Italy, 2022.
  40. Perdigão, PRL; Matos, C; Lopes, Sara M. ; Fernandes, Ana Rita; Miguel M. Lopes; Faro, R; Santana, Magda M; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Correction of ATXN3-CAG expansion in MJD patient-derived iPSC by CRISPR/Cas9 homology-directed repair.". Paper presented in 52th Meeting of the Portuguese Society of Pharmacology, 2022.
  41. Carvalhas de Almeida, Catarina; Pereira de Almeida, Luis ; Cavadas, Claudia; Perdigão, Pedro*; Álvaro, Ana Rita* . Corresponding author: Perdigão, Pedro*. "Establishing iPSC-derived neuronal cell model to study human insomnia pathophysiology". Paper presented in XVI Symposium of Experimental Biology and Biomedicine Doctoral Programme, University of Coimbra, Coimbra, Portugal, 2022.
  42. Kevin Leandro; David Rufino-Ramos; Sara Lopes; Inês M. Martins; Diana Lobo; Manuela Paoletta; Rosário Faro; Pedro Perdigão; Pereira de Almeida, Luis. "Extracellular vesicles transient delivery of CRISPR/Cas9 systems to knock-out ataxin-3 expression". Paper presented in XVII Meeting of the Portuguese Society for Neuroscience (SPN2021), Coimbra, Portugal., 2021.
  43. Perdigão, Pedro (0F1E-EFD6-AF01); Matos, Carlos A; Lopes, Sara; Fernandes, Ana Rita; Lopes, Miguel; Faro, Rosário; Santana, Magda M; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Precise and scarless correction of ATXN3-CAG expansion by CRISPR/Cas9 for the generation of patient-derived MJD isogenic iPSC". Paper presented in XVII Meeting of the Portuguese Society for Neuroscience, Coimbra, Portugal, 2021.
  44. Daniel Henriques; Moreira, Ricardo; Pedro Perdigão; Agnete Kirkeby; Luís Pereira de Almeida; Liliana Mendonça. Corresponding author: Daniel Henriques. "Generation of cerebellar neuronal progenitors for cell replacement strategies in Machado-Joseph Disease". Paper presented in XVII Meeting of the Portuguese Society for Neuroscience, 2021.
  45. Leung, A; Sacristan-Reviriego, A; Perdigão, PRL; Bainbridge, J; Michaelides, M; van der Spuy, J. "Investigation of Ataluren (PTC124) as a potential therapeutic for LCA4 in a patient-derived iPS-retinal organoid model". Paper presented in ARVO Annual Meeting 2021, 2021.
  46. Afanasyeva, T; Perdigão, PRL; Anasthasiou, D; van der Spuy, J; Cheetham, M; Roepman, R; Collin, RWJ. "Gene-corrected iPSC Lines as a Patient-Specific Model to study LCA5-Associated Retinal Disease". Paper presented in International Society for Stem Cell Research 2020 Virtual Meeting, 2020.
  47. Baptista, A.C.; Lima, N.; Perdigão, P; Taborba, S.; Faustino, Bruno; Borges, J.P.. "Exploring fiber functionalization for smart textiles". Paper presented in Materiais 2019 - XIX Congresso da Sociedade Portuguesa de Materiais & X International Symposium on Materials, 2019.
  48. Perdigao, PRL; Sacristan-Reviriego, A; Jovanovic, K; Cheetham, M; Brainbridge, J; Michaelides, M; van der Spuy, J. "Using iPSC-reprogramming and CRISPR-based genome engineering to model AIPL1-associated Leber congenital amaurosis". Paper presented in Gene Editing in Ophthalmology Meeting of Macular Society 2019, Oxford, United Kingdom, 2019.
  49. Perdigão, P; Sacristan-Reviriego, A; Jovanovic, K; Cheetham, M; Brainbridge, J; Michaelides, M; van der Spuy, J. "Using iPSC-reprogramming and CRISPR-based genome engineering to model AIPL1-associated Leber congenital amaurosis". Paper presented in UCL Institute of Ophthalmology Early Career Researcher Symposium 2019, London, United Kingdom, 2019.
  50. Perdigao, PRL; Sacristan-Reviriego, A; Jovanovic, K; Cheetham, M; Brainbridge, J; Michaelides, M; van der Spuy, J. "Using iPSC-reprogramming and CRISPR-based genome engineering to model AIPL1-associated Leber congenital amaurosis.". Paper presented in XVIIIth International Symposium on Retinal Degeneration 2018, Killarney, Ireland, 2018.
  51. Perdigao, PRL; Gaj, T; Rocha, C; Barbas III, C; Santa-Marta, M; Gonçalves, J. "Gene- therapeutic approach complementing TALE activators and suicidal vectors target and eliminate HIV latent cells.". Paper presented in FASEB Science Research Conference: Genome Engineering - Cutting-Edge Research and Applications 2016, Lisbon, Portugal, 2016.
  52. Perdigão, P; Gaj, T; Rocha, C; Barbas III, C; Santa-Marta, M; Gonçalves, J. "Gene- therapeutic approach complementing TALE activators and suicidal vectors target and eliminate HIV latent cells.". Paper presented in Portuguese Annual Meeting Ciência 2016, Lisbon, Portugal, 2016.
  53. Narciso, S; Santos, AC; Perdigão, P; Gonçalves, J. "New concept of immunotherapy for manipulation of gene expression". Paper presented in 8th iMed.ULisboa Postgraduate Students Meeting 2016, Lisbon, Portugal, 2016.
  54. Perdigão, P; Gaj, T; Barbas III, C; Santa-Marta, M; Gonçalves, J. "Reactivation of latent HIV-1 expression by engineered TALE transcription factors.". Paper presented in ESGCT and FSGT Collaborative Congress 2015, Helsinki, Finland, 2015.
  55. Perdigão, Pedro; Gaj, T; Barbas III, C; Santa-Marta, M; Gonçalves, J. "Reactivation of latent HIV-1 expression by engineered TALE transcription factors.". Paper presented in 7th iMed.UL Postgraduate Students Meeting 2015, Lisbon, Portugal, 2015.
  56. Perdigão, P; Acúrcio, R; Duarte, A; São-José, C; Santa-Marta, M; Moniz-Pereira, J; Gonçalves, J. "HIV-1 latency reactivation by bacterial molecular agents derived from opportunistic infections". Paper presented in 7th iMed.UL Postgraduate Students Meeting 2015, Lisbon, Portugal, 2015.
  57. Perdigão, P; Gaj, T; Barbas III, C; Santa-Marta, M; Gonçalves, J. "Engineered artificial transcription factors as novel tools to eliminate HIV latency". Paper presented in 6th iMed.UL Postgraduate Students Meeting 2014, Lisbon, Portugal, 2014.
  58. Perdigão, P; Santa-Marta, M; Duarte, A; Gonçalves, J. "Reactivation of latent HIV-1 by extracellular factors secreted by NI-bacteria". Paper presented in 5th iMed.UL Postgraduate Students Meeting 2013, Lisbon, Portugal, 2013.
  59. Santa-Marta, M; Perdigão, P; Gonçalves, J. "Specific elimination of HIV-1 infected cells using a Tat/Rev-dependent circuit". Paper presented in A Collaborative Solution for Tropical Diseases: The Luso-American Response. Conference on HIV/AIDS research 2011, Lisbon, Portugal, 2011.
  60. Santa-Marta, M; Gardner, R; Perdigão, P; Gonçalves, J. "Identification of the signaling pathways associated with HIV Latency". Paper presented in 5th National Meeting on Cell Signalling 2011, Coimbra, Portugal, 2011.
Journal article
  1. Leandro, Kevin; Rufino-Ramos, David; Lopes, Sara M.; Silva, Francisca Santos; Rodrigues-Santos, Paulo; Silva, Ana Carolina; Fernandes, Ana Rita; et al. "Extracellular vesicles-mediated delivery of SpCas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3". Biomaterials 331 (2026): 124119. https://doi.org/10.1016/j.biomaterials.2026.124119.
    Published • 10.1016/j.biomaterials.2026.124119
  2. Henriques, Daniel; Moreira, Ricardo; Pena, Frederico; Perdigão, PRL; Kikerby, Agnete; Pereira de Almeida, Luis ; Mendonça, Liliana. "Generation of Lineage-Specific Cerebellar Neuronal Progenitors for Cell Replacement Strategies in Cerebellar Diseases". (2026):
    Submitted
  3. Leung, Amy; Perdigão, Pedro R.L.; Sacristan-Reviriego, Almudena; Sladen, Paul E.; Aguzzi, Erika A.; Rezek, Farah.O.; Ziaka, Kalliopi; et al. "Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids". Molecular Therapy Nucleic Acids 36 4 (2025): 102777. https://doi.org/10.1016/j.omtn.2025.102777.
    Published • 10.1016/j.omtn.2025.102777
  4. Leandro, Kevin; Rufino-Ramos, David; Breyne, Koen; Di Ianni, Emilio; Lopes, Sara M.; Jorge Nobre, Rui; Kleinstiver, Benjamin P.; et al. "Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads". Advanced Drug Delivery Reviews 211 (2024): 115346. http://dx.doi.org/10.1016/j.addr.2024.115346.
    Published • 10.1016/j.addr.2024.115346
  5. Sai, Hali; Ollington, Bethany; Rezek, Farah O.; Chai, Niuzheng; Lane, Amelia; Georgiadis, Tassos; Bainbridge, James; et al. "Effective AAV-mediated gene replacement therapy in retinal organoids modelling AIPL1-associated LCA4". Molecular Therapy - Nucleic Acids (2024): 102148. http://dx.doi.org/10.1016/j.omtn.2024.102148.
    Published • 10.1016/j.omtn.2024.102148
  6. Benati, D; Leung, A; Perdigão, P; Toulis, V; van der Spuy, J; Recchia, A. "Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders". International Journal of Molecular Sciences 23 23 (2023): 15276. https://www.mdpi.com/1422-0067/23/23/15276.
    Open access • Published
  7. Martins, Mariana; Oliveira, Ana Rafaela; Martins, Solange; Vieira, José Pedro; Perdigão, Pedro; Fernandes, Ana Rita; de Almeida, Luís Pereira; et al. "A Novel Genetic Variant in MBD5 Associated with Severe Epilepsy and Intellectual Disability: Potential Implications on Neural Primary Cilia". International Journal of Molecular Sciences 24 16 (2023): 12603. http://dx.doi.org/10.3390/ijms241612603.
    Published • 10.3390/ijms241612603
  8. Afanasyeva, Tess A.V.; Athanasiou, Dimitra; Perdigao, Pedro R.L.; Whiting, Kae R.; Duijkers, Lonneke; Astuti, Galuh D.N.; Bennett, Jean; et al. "CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids". Molecular Therapy - Methods & Clinical Development 29 (2023): 522-531. http://dx.doi.org/10.1016/j.omtm.2023.05.012.
    Published • 10.1016/j.omtm.2023.05.012
  9. Pedro Perdigão; Bethany Ollington; Hali Sai; Amy Leung; Almudena Sacristan-Reviriego; Jacqueline van der Spuy. "Retinal Organoids from an AIPL1 CRISPR/Cas9 Knockout Cell Line Successfully Recapitulate the Molecular Features of LCA4 Disease". International Journal of Molecular Sciences (2023): https://www.mdpi.com/1422-0067/24/6/5912.
    10.3390/ijms24065912
  10. Rufino-Ramos, David; Leandro, K; Perdigão, Pedro (0F1E-EFD6-AF01); O'Brien, K; Pinto, MM; Santana, MM; van Solinge, TS; et al. "Extracellular communication between brain cells through functional transfer of Cre mRNA". Molecular Therapy (2023):
    In press
  11. Leung, Amy; Sacristan-Reviriego, Almudena; Perdigão, Pedro R.L.; Sai, Hali; Georgiou, Michalis; Kalitzeos, Angelos; Carr, Amanda-Jayne F.; et al. "Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis". Stem Cell Reports 17 10 (2022): 2187-2202. http://dx.doi.org/10.1016/j.stemcr.2022.08.005.
    Published • 10.1016/j.stemcr.2022.08.005
  12. 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.. "CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCs". Molecular Therapy - Nucleic Acids 26 (2021): 432-443. http://dx.doi.org/10.1016/j.omtn.2021.08.015.
    Published • 10.1016/j.omtn.2021.08.015
  13. Perdigão, Pedro R.L.; Cunha-Santos, Catarina; Barbas, Carlos F.; Santa-Marta, Mariana; Goncalves, Joao. "Protein Delivery of Cell-Penetrating Zinc-Finger Activators Stimulates Latent HIV-1-Infected Cells". Molecular Therapy - Methods & Clinical Development 18 (2020): 145-158. http://dx.doi.org/10.1016/j.omtm.2020.05.016.
    Published • 10.1016/j.omtm.2020.05.016
  14. Cunha-Santos, C; Perdigao, PRL; Oliveira, JG; Cardoso, M; Manuel, A; Taveira, N; Goncalves, J. "Inhibition of HIV replication through siRNA carried by CXCR4-targeted chimeric nanobody.". Cellular and molecular life sciences : CMLS (2019): https://doi.org/10.1007/s00018-019-03334-8.
    Published • 10.1007/s00018-019-03334-8
  15. Perdigão, Pedro; Gaj, T; Santa-Marta, M; Barbas III, C; Goncalves, J. "Reactivation of Latent HIV-1 Expression by Engineered TALE Transcription Factors.". PloS one (2016): http://europepmc.org/articles/PMC4774903.
    10.1371/journal.pone.0150037
Newspaper article
  1. Vinhas, AC; Ferreira, AC; Silva, C; Miranda, C; Henriques, D; Oliveira, D; Ramos, D; et al. ""Uma Viagem Rara"", PÚBLICO, 2020, https://www.publico.pt/2020/02/28/ciencia/fotogaleria/viagem-rara-400481.
Preprint
  1. David Rufino-Ramos; Kevin Leandro; Pedro R.L. Perdigão; Killian O’Brien; Maria Manuel Pinto; Magda M. Santana; Thomas S van Solinge; et al. "Extracellular communication between brain cells through functional transfer of Cre mRNA". 2023. https://doi.org/10.1101/2023.01.29.525937.
    10.1101/2023.01.29.525937
  2. Amy Leung; Almudena Sacristan-Reviriego; Pedro R. L. Perdigão; Hali Sai; Michalis Georgiou; Angelos Kalitzeos; Amanda-Jayne F. Carr; et al. "Translational readthrough as a potential therapeutic for AIPL1-associated Leber Congenital Amaurosis in a patient-derived iPSC-retinal organoid model". 2021. https://doi.org/10.1101/2021.12.17.473147.
    10.1101/2021.12.17.473147
Thesis / Dissertation
  1. Perdigão, Pedro. "Gene-targeted strategies to eliminate HIV latent cells through synthetic activators and suicide lentivectors". PhD, Universidade de Lisboa Faculdade de Farmácia, 2017. http://hdl.handle.net/10451/29864.
  2. Perdigão, Pedro. "Specific elimination of HIV-1 infected cells using a Tat/Rev-dependent circuit". Master, Universidade de Lisboa Faculdade de Farmácia, 2011. http://hdl.handle.net/10451/29864.
Working paper
  1. Kevin Leandro; David Rufino-Ramos; Sara M. Lopes; Paulo Santos; Ana Carolina Silva; Ana Rita Fernandes; Carina Henriques; et al. Corresponding author: Luis Pereira de Almeida. 2025. "Extracellular vesicles mediated delivery of CRISPR-Cas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3 (under revision in Biomaterials)".

Intellectual property

Provisional application for patent
  1. 2025. "RNA-TARGETING CRISPR/CAS13 FOR ALLELE-SPECIFIC KNOCKDOWN".
    Protected
  2. 2025. "METHODS FOR PRODUCING A REPORTER NEURONAL MODEL AND USES THEREOF".
    Protected
  3. 2025. "ATXN3-TARGETING CRISPR BASE EDITING SEQUENCES, METHODS AND USES THEREOF".
    Protected
Activities

Oral presentation

Presentation title Event name
Host (Event location)
2026/04/16 Engineering Isogenic iPSC Disease Models with Precision Genome Editing ESGCT Spring School 2026
European Society for Gene and Cell Therapy (Coimbra, Portugal)
2026/02/05 Establishment of a scalable neuronal reporter model of Machado-Joseph disease based on gene-edited patient cells as a platform for high-throughput drug repurposing 56ª Reunião da Sociedade Portuguesa de Farmacologia
Sociedade Portuguesa de Farmacologia (Coimbra, Portugal)
2026/02/05 ENGINEERING RNA-TARGETING CRISPR/CAS13 FOR ALLELE-SPECIFIC ATXN3 KNOCKDOWN FOR TREATMENT OF SPINOCEREBELLAR ATAXIA TYPE 3 56ª Reunião da Sociedade Portuguesa de Farmacologia
Sociedade Portuguesa de Farmacologia (Coimbra, Portugal)
2025/11/20 Establishment of a scalable neuronal reporter model of Machado-Joseph disease based on gene-edited patient cells as a platform for high-throughput drug repurposing 29º Reunião Anual da Sociedade Portuguesa de Genética Humana
Sociedade Portuguesa de Genética Humana (Coimbra, Portugal)
2022/11/17 Modelling Machado-Joseph Disease in Patient-Derived Isogenic Lines by CRISPR/Cas9-Mediated Correction of ATXN3-CAG Expansion 26th Annual SPGH Meeting
Portuguese Society of Human Genetics (Coimbra, Portugal)
2022/02/10 Correction of ATXN3-CAG Expansion in MJD Patient-Derived iPSC by CRISPR/Cas9 Homology-Directed Repair LII SPF Meeting
Portuguese Society of Pharmacology (Porto, Portugal)
2021/05/24 Genome Editing 2.0: CRISPR-Base Editors CNC course in Principles and Practice in Drug Development
Center for Neuroscience and Cell Biology (Coimbra, Portugal)
2021/04/21 Genome editing 2.0: CRISPR-base editors PDBEB Course - Advanced Therapies
Center for Neuroscience and Cell Biology (Coimbra, Portugal)
2020/11/24 Edição Genética: o novo motor evolutivo? Shot de Ciência
Núcleo de Estudantes de Biologia da Associação Académica de Coimbra (NEB/AAC)
2020/10/07 Genome editing methods in the context of Machado-Joseph Disease Curso de Verão – Metodologias de Investigação Científica
Instituto de Investigação Interdisciplinar da Universidade de Coimbra (iiiUC) (Coimbra, Portugal)
2017 Gene-therapeutic approach complementing TALE activators and suicidal vectors target and eliminate HIV latent cells Portuguese Annual Meeting Ciência 2017, Lisbon, Portugal
Fundação para a Ciência e a Tecnologia (Lisbon, Portugal)
2016 Gene-therapeutic approach complementing TALE activators and suicidal vectors target and eliminate HIV latent cells 8th iMed.UL Postgraduate Students Meeting 2016, Lisboa, Portugal
Research Institute for Medicines (iMed.ULisboa) (Lisbon, Portugal)
2016 Gene-therapeutic approach complementing TALE activators and suicidal vectors target and eliminate HIV latent cells FASEB Science Research Conference: Genome Engineering - Cutting-Edge Research and Applications 2016, Lisbon, Portugal
Federation of American Societies For Experimental Biology (Lisbon, Portugal)
2015 Specific elimination of latent HIV infected cells through a shock and kill suicidal gene therapy 8th Biennal Congress of the Spanish Society of Gene and Cell Therapy 2015, San Sebastian, Spain
Spanish Society of Gene and Cell Therapy (San Sebastian, Spain)

Supervision

Thesis Title
Role		 Degree Subject (Type)
Institution / Organization
2024/05/01 - Current Establishing a Rapid and Robust Human Neuronal Model of Machado-Joseph Disease from Patient-Derived Gene-Edited Isogenic iPSC Lines
Supervisor
 Biotecnologia Farmacêutica (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2024/03/25 - Current Engineering RNA-targeting CRISPR/Cas13 for modulation of ATXN3 pre-mRNA splicing as a novel treatment of spinocerebellar ataxia type 3
Supervisor
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2021/10/01 - Current Assessing the impact of ATXN3 silencing in the context of a human model of Machado-Joseph Disease
Co-supervisor
 Ciências Farmacêuticas (PhD)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2021/09/01 - Current High-content screening of a novel therapy for Machado-Joseph disease in reporter gene-edited induced pluripotentstem cells
Co-supervisor
 Ciências Farmacêuticas (PhD)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2021/07/05 - Current Modulation of ATXN3 splicing by CRISPR-base editors for treatment of Machado-Joseph Disease in patient-derived cellular models
Supervisor of Mário Barreira
 Biotecnologia Farmacêutica (Master)
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Universidade de Coimbra Faculdade de Farmácia, Portugal
2023/09/01 - 2024/10/22 Engineering RNA-Targeting CRISPR/Cas13 for Allele-Specific ATXN3 Knockdown as a Novel Treatment for Spinocerebellar Ataxia Type 3
Supervisor
 Biotecnologia Farmacêutica (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2022/09/01 - 2023/10/16 Engineering CRISPR base editors to rescue Machado-Joseph Disease in a patient-derived neuronal model.
Supervisor of Miguel Dias
 Biotecnologia Farmacêutica (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2022/09/01 - 2022/09/30 Gene Therapy for Spinocerebellar Ataxia Type 3 (Supervisor in the scope of "Verão com Ciência 2022" training action coordinated by Fundação pela Ciência e Tecnologia)
Supervisor of Miguel Dias
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2021/02/16 - 2021/08/03 Design and validation of AAV constructs for delivery of CRISPR-base editors to rescue Machado-Joseph Disease (Supervisor for the "Projecto em Biologia Aplicada" course integrated in the Biologia bachelor degree)
Supervisor of Miguel Lucas
 Biologia (Scientific initiation)
Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
2020/08/01 - 2020/10/31 Genome editing methods for the generation and characterization of isogenic models of neurologic disease (Supervisor in the scope of "Verão com Ciência 2020" training action coordinated by Fundação pela Ciência e Tecnologia)
Supervisor of Ana Rita Fernandes
 Verão com Ciência (Scientific initiation)
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Event participation

Activity description
Type of event		 Event name
Institution / Organization
2022/11/14 - 2022/11/18 Quantitative Fluorescence Microscopy Course
Workshop
 VI Quantitative Fluorescence Microscopy Course
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2020/11/09 - 2020/11/20 Animal Experimentation Course IV Modular Training in Laboratory Animal Science
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2019 - 2019 Course in Creativity in research
Workshop
University College London Human Resources, United Kingdom
2018/12/03 - 2018/12/03 Course in Quality papers
Workshop
University College London Human Resources, United Kingdom
2018/06/14 - 2018/06/14 UCL Institute of Ophthalmology Early Career Researcher Symposium 2018
Symposium
University College London Institute of Ophthalmology, United Kingdom
2017/01/19 - 2017/01/19 Workshop Elsevier in How to write a great research paper and get it accepted by a good journal
Workshop
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
2015/07/02 - 2015/07/02 Workshop Hands-On in Preventive maintenance and calibration of micropipettes
Workshop
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
2015/01/28 - 2015/01/28 Workshop in Design of experiments in pharmaceutical sciences research
Workshop
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
2014/03/13 - 2014/03/14 Workshop in BSL2 and BSL3
Workshop
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
2013/10/23 - 2013/10/23 Workshop in Grant writing: tips and thoughts
Workshop
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
2012/01/18 - 2012/01/18 Workshop in Preparation of quality figures for publication
Workshop
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal

Jury of academic degree

Topic
Role		 Candidate name (Type of degree)
Institution / Organization
2024/09/11 “Biodegradable Polymeric Nanoparticles for the Delivery of Gene Editing Tools”
(Thesis) Main arguer
 Anna Anjo-Stefanidis (Master)
Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
2024/03/18 Insights into AAV promoter activity and helper virus components interactions
(Thesis) Main arguer
 Júlia Reis de Lidon Brandão Guerra (Master)
Universidade de Lisboa Faculdade de Ciências, Portugal
2023/04/11 Assessing the Role of Nucleocytoplasmic Transport Mechanisms in Machado-Joseph Disease Pathogenesis
(Thesis) Arguer
 Maria Inês Morgado Martins (PhD)
Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal
2020/12/11 Biodistribution Analysis of Striatal and Cerebellar Administration of Modified Adeno-Associated Viral Vectors in Normal Mice
(Thesis) Main arguer
 Inês Torquato Afonso (Master)
Universidade do Algarve, Portugal
2020/09/22 Edição de bases: o futuro da edição de genes?
(Thesis) Main arguer
 Irina Marques da Silva Fernandes (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2019/09/17 Terapia Génica na Diabetes Mellitus tipo 1
(Thesis) Main arguer
 Marta Salvado Abreu Castelo Branco (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal

Course / Discipline taught

Academic session Degree Subject (Type) Institution / Organization
2024/11/06 - 2024/11/06 Invited Lecturer for the lecture series “DCV Talks” Life Sciences Department, Faculty of Science and Technology, University of Coimbra “CRISPR-Based Therapeutic Approaches for Machado-Joseph Disease” (Outros) Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
2023/12/11 - 2023/12/11 Invited Lecturer for the Gene Therapy course from the BSc Biomedical Sciences Faculty of Medicine, University of Algarve “Harnessing CRISPR gene editing platforms to model or treat Machado-Joseph Disease” Gene Therapy (Licenciatura) Universidade do Algarve Faculdade de Medicina e Ciências Biomédicas, Portugal
2021/03/16 - 2021/03/16 Lecture in "Genome editing 2.0: CRISPR-base editors" (Discipline- Human Molecular Genetics) Análises Clínicas (Mestrado integrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2020/04/04 - 2020/04/04 Lecture in "Genome Editing and Induced Pluripotent Stem Cells" (Discipline- Tissue Engineering and Molecular Therapy) Biotecnologia Farmacêutica (Mestrado integrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2016/05/03 - 2016/05/03 Lecture in "Genome Editing: applications for human gene therapy" (Discipline- Biotecnology Pharmaceutics) Ciências Biofarmacêuticas (Mestrado integrado) Universidade de Lisboa Faculdade de Farmácia, Portugal

Mentoring / Tutoring

Topic Student name
2024/04/01 - Current Mentor of PhD candidate Maria Luísa Amado "Exploring the contribution of homeostatic plasticity mechanisms in the pathogenesis of autism spectrum disorder" Maria Luísa Amado
2021/02 - Current Mentor of PhD candidate Catarina Carvalhas-Almeida "Establishing iPSC-derived neuronal cell model to study human insomniapathophysiology" Catarina Carvalhas-Almeida
2020/01 - 2022/04 Mentor of PhD candidate Ana Catarina Pereira in "Establishing iPSC-derived models to investigate mechanisms of bipolar disease” Ana Catarina Pereira
2019/01 - 2019/06 Mentor of PhD candidate Tess Afanasyeva in "CRISPR/Cas9-based genome editing methods for the correction of mutations associated with Leber Congential Amaurosis type 5 in patient-derived iPSCs" Tess Afanasyeva
2019/01 - 2019/06 Mentor of PhD candidate Paul Sladen in "CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H in dominant optic atrophy patient-derived iPSCs" Paul Sladen
2016/09 - 2017/06 Mentor of MSc candidate Miguel Cardoso in "Novel Strategy to Detect and Localize Latent HIV Reservoirs" Miguel Cardoso
2015/01 - 2016/12 Mentor of MSc researcher Rita Acúrcio in "Reactivation of latent HIV-1 by nocosomial bacterial infections" Rita Acúrcio
2015/09 - 2016/09 Mentor of MSc candidate Ana Sofia Narciso in "New Concept of Immunotherapy for Gene Manipulation" Ana Sofia Narciso
2015/09 - 2016/09 Mentor of MSc candidate Maria Inês Gonçalves in "O papel do DNA-PKcs na transcrição mediada pelo promotor LTR do HIV-1" Maria Inês Gonçalves
2012/09 - 2013/09 Mentor of MSc candidate Vasco Gonçalves in "Targeting Membrane Proteins of Latently Infected CD4+ T Cells Providing a Starting Point to the Eradication of HIV­1" Vasco Gonçalves

Other jury / evaluation

Activity description Institution / Organization
2024/03/11 - Current Jury member (Effective) for assignment of 5 MSc Researcher fellowships Reference: CiNTech - Technological Hub for Innovation, Translation and Industrialization of Complex Injectable Drugs” (7131) financed by the PRR - Recovery and Resilience Plan and by the Next Generation EU European Funds) Center for Neuroscience and Cell Biology, University of Coimbra Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2024/03/01 - Current Jury member (President) for assignment of an MSc Researcher fellowships Reference: “Engineering RNA-Targeting CRISPR/Cas13 for the Treatment of Spinocerebellar Ataxia Type 3” (25124), financed by AFM-Téléthon Center for Neuroscience and Cell Biology, University of Coimbra Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2020/09/27 - 2020/09/27 Participation as a jury member for the assignment of an MSc researcher fellowship (reference: BIM-PAC CANCEL_STEM/2020/06) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Distinctions

Award

2022 Best Oral Communication in Basic Research, 26th Annual SPGH Meeting
Sociedade Portuguesa de Genética Humana, Portugal
2018 Travel Award - XVIIIth International Symposium on Retinal Degeneration, Killarney, Ireland
Foundation Fighting Blindness, Canada
2016 Top Abstract Award (Oral communication) - FASEB Science Research Conference: Genome Engineering - Cutting-Edge Research and Applications, Lisbon, Portugal
Federation of American Societies for Experimental Biology, United States
2016 Best oral communication – 8th iMed.UL Postgraduate Students Meeting, Lisboa, Portugal
Universidade de Lisboa Instituto de Investigação do Medicamento, Portugal
2015 Best oral communication – 8th Biennal Congress of the Spanish Society of Gene and Cell Therapy, San Sebastian, Spain
Spanish Society of Gene and Cell Therapy, Spain
2011 PhD Fellowship (SFRH/BD/81941/2011)
Fundação para a Ciência e a Tecnologia, Portugal