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Sara M Lopes (PharmD, PhD) is a junior researcher at the Gene and Stem Cell Therapies for the Brain Group at the Center for Neuroscience and Cell Biology (CNC), University of Coimbra. She got her PhD in 2021, where she acquired a vast experience on the design and construction of sequence-specific nucleases (with expertise in TALENs and CRISPR systems), viral vectors for therapy (lentivirus and adeno-associated virus) and pre-clinical models of the disease. Sara is the author of 7 articles in scientific journals, 2 book chapters and of 2 provisional patent applications. She has an active participation in both national and international scientific meetings, being the author of a total of 36 oral communications and 34 poster presentations. The work that she has developed was awarded twice with the Best Work in Basic Research, attributed by the Portuguese Society of Movement Disorders in 2018 and by the Portuguese Society of Human Genetics in 2020. More recently she participated in entrepreneurship programmes, having received the bronze award (10.000€) from the EIT health DPhil PhD transition Fellowship for the project ExoEditor (2017) and the first prize (20.000€) of the innovation programme in neuroscience (Building Tomorrow Together) for the project ExoTreat, an initiative from Roche (2020).
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Identification

Personal identification

Full name
Sara Isabel Monteiro Lopes

Citation names

  • Lopes, Sara M.
  • Sara M. Lopes
  • Lopes, Sara
  • Sara Lopes
  • Lopes, S. M.
  • S. M. Lopes

Author identifiers

Ciência ID
D715-EE08-5DD9
ORCID iD
0000-0002-4322-8529

Knowledge fields

  • Medical and Health Sciences - Basic Medicine - Pharmacology and pharmacy
  • Medical and Health Sciences - Medical Biotechnology - Health-Related Biotechnology
  • Medical and Health Sciences - Medical Biotechnology - Technologies Involving the Manipulation of Cells, Tissues, Organs or the Whole Organism
  • Medical and Health Sciences - Medical Biotechnology - Gene-based Diagnostics and Therapeutic Interventions
  • Medical and Health Sciences - Basic Medicine - Neurosciences
  • Medical and Health Sciences - Basic Medicine - Human Genetics
  • Natural sciences - Biological Sciences - Cell Biology
  • Natural sciences - Biological Sciences - Molecular Biology
  • Natural sciences - Biological Sciences - Microbiology

Languages

Language Speaking Reading Writing Listening Peer-review
Portuguese (Mother tongue)
English Proficiency (C2) Proficiency (C2) Proficiency (C2) Proficiency (C2)
Spanish; Castilian Upper intermediate (B2) Advanced (C1) Upper intermediate (B2) Advanced (C1)
French Beginner (A1) Intermediate (B1) Beginner (A1) Elementary (A2)
Education
Degree Classification
2021/10/21
Concluded
 Biologia Experimental e Biomedicina (Doutoramento)
Major in Biotecnologia e Saúde
Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
"Gene editing technologies to Machado-Joseph disease" (THESIS/DISSERTATION)
 Louvor e distinção por unanimidade
2010/09/24
Concluded
 Ciências Farmacêuticas (Mestrado integrado)
Universidade de Coimbra Faculdade de Farmácia, Portugal
"Antidepressivos e Neurogénese" (THESIS/DISSERTATION)
 15
Affiliation

Science

Category
Host institution 		Employer
2023/06 - Current Contracted Researcher (Research) Universidade de Coimbra, Portugal
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
(...)
2022/01/01 - 2023/04 Postdoc (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2019/11/18 - 2021/12/31 Researcher (Research) Universidade de Coimbra, Portugal
Vectors and Gene Therapy Group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal
2017/08/01 - 2019/09/30 Researcher (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Vectors and Gene Therapy Group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal
2016/10/01 - 2017/06/30 Researcher (Research) Universidade de Coimbra, Portugal
Vectors and Gene Therapy Group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal
2016/04/15 - 2016/09/15 Researcher (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Vectors and Gene Therapy Group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal
2015/10/01 - 2015/12/31 Researcher (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Vectors and Gene Therapy Group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal
2010/09/01 - 2011/08/31 Researcher (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Redox Biology and Brain Sensing Group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal
2009/10 - 2010/01 Research Trainee (Research) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Neuroendocrinology and Ageing group, Center for Neuroscience and Cell Biology, University of Coimbra, Portugal

Others

Category
Host institution 		Employer
2012/10/18 - 2012/11/19 Graduate Student Researcher Broad Institute, United States
Projects

Grant

Designation Funders
2023/09/01 - 2026/08/31 NEURODIET - Molecular Mechanisms of Dietary Intervention on Neurodegeneration (PT001)
JPND/0001/2022
10.54499/JPND/0001/2022
Researcher
Universidade de Coimbra, Portugal

Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Fundação para a Ciência e a Tecnologia
Ongoing
2022/01/01 - 2023/06/30 Genome editing to rescue Machado-Joseph disease by alternative splicing
EXPL/MED-NEU/0936/2021
Universidade de Coimbra, Portugal

Universidade de Coimbra Instituto de Investigação Clínica e Biomédica de Coimbra, Portugal
Fundação para a Ciência e a Tecnologia
Concluded
2022/11/01 - 2023/06/29 Fighting Sars-CoV-2 with viral vectors
CENTRO-01-01D2-FEDER000002
Post-doc Fellow
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Universidade de Coimbra, Portugal
Universidade de Coimbra
Concluded
2019/11/18 - 2021/12/31 BrainHealth 2020: Detecção precoce, neuro-modulação e terapias avançadas para neuropatologias
CENTRO-01-0145-FEDER-000008
Research Fellow
CNC.IBILI, Portugal
 Centro 2020 (Programa Operacional da Região Centro)
Concluded
2010/09/01 - 2011/08/31 In vivo dynamics of nitric oxide in the brain: regulation of cellular respiration and implications for Alzheimer´s disease and aging
PTDC/SAU-NEU/103538/2008
Research Fellow
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Associação para a Inovação e Desenvolvimento da FCT
Concluded

Contract

Designation Funders
2023/06/30 - Current CiNTech - Technological Hub for Innovation, Translation and Industrialization of Complex Injectable Drugs
C644865576-00000005
Researcher
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Universidade de Coimbra, Portugal
Universidade de Coimbra
Ongoing
2019 - Current Targeting mutant ATXN3 for the treatment of Spinocerebellar Ataxia 3 (SCA3) using allele-specific ASOs
N.A
Researcher
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Wave Life Sciences
Ongoing
2022/01 - 2022/10 Collaboration agreement - F. Hoffmann - La Roche Ltd (Roche Group)
n.a
Post-doc Fellow
Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
F Hoffmann-La Roche AG

F Hoffmann-La Roche AG Research and Development Division
2015/01/01 - 2019/12/31 CNC.IBILI
UID/NEU/04539/2013
UID/NEU/04539/2019
Researcher
CNC.IBILI, Portugal
 Associação para a Inovação e Desenvolvimento da FCT
Concluded
2017/08/01 - 2019/09/30 CutCAGs: Gene repair and repression strategies to alleviate Machado-Joseph disease
PTDC/NEU-NMC/0084/2014
POCI-01-0145-FEDER-016719
Research Fellow
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Associação para a Inovação e Desenvolvimento da FCT
Concluded
2016/10/01 - 2017/06/30 Research in stem cells and Machado Joseph disease
DPA-56-16-388
Research Fellow
Universidade de Coimbra, Portugal

Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Concluded
2016/04/15 - 2016/09/15 Towards the understanding of pathological protein processing and toxicity in Machado-Joseph Disease
E-Rare4/0003/2012
Research Fellow
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Concluded
2015/10/01 - 2015/12/31 Cabaletta@CNC
n.a.
Research Fellow
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 BioBlast Pharma, Ltda
Concluded
2011/10/01 - 2015/09/30 Gene Editing Technologies to Machado-Joseph Disease
SFRH/BD/51673/2011
PhD Student Fellow
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Associação para a Inovação e Desenvolvimento da FCT
Concluded
2013 - 2014 Strategic Project - LA 1 - 2013-2014
PEst-C/SAU/LA0001/2013
Researcher
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Associação para a Inovação e Desenvolvimento da FCT
Concluded
2011 - 2012 Strategic project - LA 1 - 2011-2012
PEst-C/SAU/LA0001/2011
Researcher
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
 Associação para a Inovação e Desenvolvimento da FCT
Concluded
Outputs

Publications

Book chapter
  1. Lopes, Sara Monteiro; Pereira de Almeida, Luis. "Gene editing and central nervous system regeneration". In Handbook of Innovations in Central Nervous System Regenerative Medicine, edited by Antonio J. Salgado, 399-433. Elsevier, 2020.
    Published • 10.1016/b978-0-12-818084-6.00011-8
  2. Matos, Carlos A.; Carmona, Vítor; Vijayakumar, Udaya-Geetha; Lopes, Sara; Albuquerque, Patrícia; Conceição, Mariana; Nobre, Rui Jorge; Nóbrega, Clévio; Pereira de Almeida, Luis. "Gene Therapies for Polyglutamine Diseases". In Polyglutamine Disorders, edited by Nóbrega, Clévio; de Almeida, Luís Pereira, 395-438. Springer International Publishing, 2018.
    Published • 10.1007/978-3-319-71779-1_20
Conference abstract
  1. Lopes, Sara M. ; Miguel M. Lopes; Nobre, Rui Jorge; Vasconcelos-Ferreira, Ana; Pereira, Dina; Fernandes, Ana Rita; Barros, Inês; et al. Corresponding author: Pereira de Almeida, Luis. "Targeting the ATXN3 gene with CRISPR-Cas9 for the therapy of Machado-Joseph disease". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, 2024.
    Published
  2. Lopes, Sara M. ; Miguel M. Lopes; Rui Nobre; Clévio Nóbrega; Matos, Carlos A; Ana Vasconcelos-Ferreira; Neville Sanjana; et al. Corresponding author: Pereira de Almeida, Luis. "Permanent inactivation of the ATXN3 gene: a possible therapeutic approach for Machado-Joseph disease". Paper presented in 24th Annual Meeting of the Portuguese Society of Human Genetics (SPGH), Virtual Conference, 2021.
    Published
Conference poster
  1. 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.
  2. Barros, Inês; Gonzaga, Daniela; Lobo, Diana Duarte; Lopes, Sara; Martins, Inês; Silva, Ana Carolina; Pereira de Almeida, L; Miranda, Catarina. Corresponding author: Miranda, Catarina. "The Guardians of Balance: Mesenchymal Stem Cells as a Promising Strategy to Restore Blood-brain Barrier Integrity in Spinocerebellar Ataxia Type 3.". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  3. Magda M Santana; D. Adão; De Luca, Pasqualino; R. Martins; Fernandes, Ana Rita; M. M. Pinto; Lopes, Sara M. ; Pereira de Almeida, Luis . Corresponding author: Pereira de Almeida, Luis. "Neural induction and neuronal maturation of induced pluripotent stem cell is compromised in Machado-Joseph Disease lines". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  4. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Miguel M. Lopes; Pereira, Dina; Duarte, Sonia P.; et al. "Allele-specific silencing of mutant ATXN3 rescues motor deficits and neuropathology in a severely impaired mouse model of spinocerebellar ataxia type 3 (SCA3) upon intra-cisterna magna administration.". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  5. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Pereira, Dina; Duarte, Sonia P.; Lopes, Sara M. ; Miguel M. Lopes; et al. "Investigating routes of administration and AAV serotypes for development of a gene-based therapy for spinocerebellar ataxia type 3 (SCA3)". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  6. 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.
  7. 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.
  8. Lopes, Sara Monteiro; Miguel M. Lopes; Nobre, Rui Jorge; Vasconcelos-Ferreira, Ana; Pereira, Dina; Fernandes, Ana Rita; Barros, Maria Inês; et al. Corresponding author: Pereira de Almeida, Luis. "Targeting the ATXN3 gene with CRISPR-Cas9 for the therapy of Machado-Joseph disease". Paper presented in 30th Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT), Brussels, Belgium, 2023.
  9. De Luca, Pasqualino; Adão, Diana; Martins, Rita; Fernandes, Ana Rita; Pinto, Maria M; Lopes, Sara M. ; Magda M Santana; Pereira de Almeida, Luis . Corresponding author: Pereira de Almeida, Luis. "Neuronal maturation of induced pluripotent stem cell is altered in Machado Joseph-Disease cell lines". Paper presented in ETRS & SPCE-TC joint meeting 2023, Coimbra, Portugal, 2023.
  10. Leandro, Kevin; Rufino-Ramos, David; Lopes, Sara M. ; Pasqua, Carlo; Santos, Paulo; Silva, Ana Carolina; Lobo, Diana Duarte; Pereira de Almeida, Luis. "Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease". Paper presented in 2023 CAG Triplet Repeat Disorder -Gordon Research Conference, West Dover, Vermont, United States of America, 2023.
  11. Pedro Perdigão; Miguel Dias; Lopes, Sara M. ; Rita Fernandes; Frederico Pena; Miguel M. Lopes; Rui Nobre; Thomas Gaj; Luis Pereira de Almeida. "Engineering CRISPR-Based Editors for Treatment of Machado-Joseph Disease". Paper presented in American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, 2023.
  12. Ribeiro, Rodrigo; Pereira, Dina; Reis, Tiago; Lopes, Sara M. ; Ferreira-Marques, Marisa; Fernandes, Rita; Gaspar, Laetitia S; et al. "The Circadian Clock is Disrupted in the YAC Machado-Joseph Disease Mouse Model". Paper presented in BEB Day 2023 - Doctoral Programme in Experimental Biology and Biomedicine (PDBEB) 17th symposium, 2023.
  13. Miguel M. Lopes; J. Paysan; J. Rino; Lopes, Sara M. ; Pereira de Almeida, Luis ; L. Cortes; R. J. Nobre. "Biodistribution analysis of AAV vectors in the brain using tissue clearing coupled to light-sheet microscopy". Paper presented in ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, Edinburg, Scotland, 2022.
  14. Perdigão, P; Matos, Carlos A; Lopes, Sara; Fernandes, Ana Rita; Miguel M. Lopes; 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.
  15. 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.
  16. Silva, Ana Carolina; Lobo, Diana Duarte; Henriques, Carina; Martins, M. I.; Sara M. Lopes; Duarte, Sonia, P. ; Wagner, E. ; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Silencing of mutant ATXN3 through antisense oligonucleotides rescues molecular and neuropathological features in Machado-Joseph disease mice". Paper presented in 29th Congress of the European Society for Gene and Cell Therapy (ESGCT) in collaboration with BSGCT; Edinburgh, Scotland, United Kingdom, 2022.
  17. Lopes, Sara M. ; Miguel M. Lopes; Rui J. Nobre; Clévio Nóbrega; Carlos A. Matos; Ana Vasconcelos-Ferreira; Dina Pereira; et al. Corresponding author: Pereira de Almeida, L. "Engineered nucleases targeting the ATXN3 gene for the therapy of Machado-Joseph disease". Paper presented in 27th European Society for Animal Cell Technology Meeting (ESACT2022), Lisbon, Portugal, 2022.
  18. Ana Carolina Silva; Diana D. Lobo; Carina Henriques; Inês M. Martins; Sara M. Lopes* (D715-EE08-5DD9); Sónia P. Duarte; Elizabeth Wagner; Rui Jorge Nobre; Pereira de Almeida, Luis. "Antisense oligonucleotide mediated allele-specific silencing of mutant ATXN3 improves disease features in mouse models of Machado-Joseph disease". Paper presented in XVII Meeting of the Portuguese Society for Neuroscience (SPN2021), Coimbra, Portugal., 2021.
  19. 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.
  20. Pedro Perdigão; Carlos Matos; Sara Lopes; Rita Fernandes; Miguel M. Lopes; Rosário Faro; Magda Santana; Rui Nobre; 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 (SPN2021), Coimbra, Portugal., 2021.
  21. Silva, Ana Carolina; Lobo, Diana Duarte; Henriques, Carina; Martins, Inês M.; Lopes, Sara M. ; Duarte, Sónia P; Wagner, Elizabeth; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Allele-specific silencing of Machado-Joseph disease using antisense oligonucleotide therapeutics". Paper presented in 1st Symposium on Oligonucleotide Technologies and Therapeutics in Portugal (OTP2021), 2021.
  22. Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes (D715-EE08-5DD9); Rui J. Nobre; Luísa Cortes; Pereira de Almeida, Luis. "Whole-Brain Analysis of Adeno-Associated Virus (AAV) Biodistribution with Single-Cell Resolution by Tissue Clearing and Light-Sheet Microscopy.". Paper presented in 1st Annual Meeting in Pharmaceutical Sciences. Coimbra, Portugal., 2019.
  23. Sara Lopes* (D715-EE08-5DD9); Carlos Matos*; André Conceição; Frederico Pena; Miguel M. Lopes; Rui Nobre; Clévio Nóbrega; et al. "CRISPR-Cas9 systems targeting ATXN3 as therapeutic approaches for Machado-Joseph Disease.". Paper presented in International Ataxia Research Conference (IARC 2019), Washington, DC. USA, 2019.
  24. Ana Ferreira; Inês Martins; Diana Lobo; Pereira, Dina; Miguel M. Lopes; Sara Lopes (D715-EE08-5DD9); Rosário Faro; et al. "Reinstating levels of dysregulated autophagy-associated transcripts alleviates neuropathology and motor impairments of mouse models of Machado-Joseph Disease.". Paper presented in International Ataxia Research Conference (IARC 2019). Washington, DC. USA., 2019.
  25. Sara Lopes* (D715-EE08-5DD9); Carlos Matos*; André Conceição; Frederico Pena; Miguel M. Lopes; Rui Nobre; Clévio Nóbrega; et al. "CRISPR-Cas9 systems targeting ATXN3 as therapeutic approaches for Machado-Joseph Disease.". Paper presented in 2019 International Machado-Joseph Research Disease Conference. Washington, DC. USA., 2019.
  26. Ana Ferreira; Inês Martins; Diana Lobo; Pereira, Dina; Miguel M. Lopes; Sara Lopes (D715-EE08-5DD9); Rosário Faro; et al. "Reinstating levels of dysregulated autophagy-associated transcripts alleviates neuropathology and motor impairments of mouse models of Machado-Joseph Disease.". Paper presented in 2019 International Machado-Joseph Research Disease Conference. Washington, DC. USA., 2019.
  27. Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes (D715-EE08-5DD9); Rui J. Nobre; Luísa Cortes; Pereira de Almeida, Luis. "Whole-Brain Analysis of Adeno-Associated Virus (AAV) Biodistribution with Single-Cell Resolution by Tissue Clearing and Light-Sheet Microscopy.". Paper presented in EMBO | EMBL Symposium: Seeing Is Believing – Imaging the Molecular Processes of Life. Heidelberg, Germany., 2019.
  28. Carlos A. Matos; André Conceição; Sara Lopes (D715-EE08-5DD9); Pereira de Almeida, Luis . Corresponding author: Pereira de Almeida, Luis. "CRISPR-Cas9 as a tool for silencing and correcting ATXN3, the gene causing Machado-Joseph disease.". Paper presented in XVI Meeting of the Portuguese Society for Neuroscience (SPN 2019). Lisbon, Portugal., 2019.
  29. Carlos A. Matos; André Conceição; Frederico Pena; Sara Lopes (D715-EE08-5DD9); Sónia Duarte; Catarina Miranda; Pereira de Almeida, Luis . Corresponding author: Pereira de Almeida, Luis. "Transcriptional repression of mutant human ATXN3 using CRISPR interference mitigates the pathological phenotype of an in vivo Machado-Joseph disease model.". Paper presented in XVI Meeting of the Portuguese Society for Neuroscience (SPN 2019). Lisbon, Portugal., 2019.
  30. Ana Vasconcelos-Ferreira; Inês Morgado Martins; Diana Lobo; Pereira, Dina; Miguel M. Lopes; Sara Lopes (D715-EE08-5DD9); Rosário Faro; et al. "Reinstating levels of dysregulated autophagy-associated transcripts alleviates neuropathology and motor impairments of mouse models of Machado-Joseph disease.". Paper presented in XVI Meeting of the Portuguese Society for Neuroscience (SPN 2019). Lisbon, Portugal, 2019.
  31. Matos, C.A.; Pena, F.; Conceição, A.; Lopes, Sara M. ; Duarte, S.; Miranda, C.; Pereira de Almeida, Luis. "CRISPR-Cas9-mediated pre-transcriptional silencing of mutant ATXN3 decreases motor impairments of an in vivo Machado-Joseph disease model.". Paper presented in 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC, USA., 2019.
  32. Lopes, S. M. (D715-EE08-5DD9); Nobre, R.; Nóbrega, C.; Lopes, M.M; Matos, C.A.; Sanjana, N.; Hsu, P.; et al. "TALEN and CRISPR gene-editing for treatment of Machado-Joseph disease.". Paper presented in 2nd International Ataxia Research Conference (IARC 2017). Pisa, Italy., 2017.
  33. Santana, M.M.*; Lopes, S. M.* (D715-EE08-5DD9); Onofre, I.; Álvaro, A.R.; Di Donato, R.; Pereira de Almeida, Luis ; *Equally contributed. "Generation of Machado-Joseph disease induced pluripotent cell lines and isogenic controls, using the CRISPR/Cas9 technology.". Paper presented in 2nd International Ataxia Research Conference (IARC 2017). Pisa, Italy., 2017.
  34. Matos, C.A.; Lopes, S. (D715-EE08-5DD9); Pereira de Almeida, Luis. "CRISPR-Cas9 as a tool for Machado-Joseph disease treatment and animal model generation.". Paper presented in EMBO Practical Course on Mouse Genome Engineering. Max Planck Institute of Molecular Cell Biology and Genetics (MPI-CBG), Dresden, Germany., 2017.
  35. Onofre, Isabel; Mendonça, Nuno; Lopes, Sara (D715-EE08-5DD9); Nobre, Rui; Barbosa, Joana; Carreira, Isabel M; Carvalho, Lina; et al. "Characterization of a human Machado-Joseph Disease neuronal cell model derived from induced pluripotent stem cells.". Paper presented in 9th Annual meeting of the Portuguese Society for Stem Cells and Cell Therapies. Braga, Portugal., 2015.
  36. Onofre, Isabel; Mendonça, Nuno; Lopes, Sara (D715-EE08-5DD9); Nobre, Rui; Barbosa, Joana; Carreira, Isabel M.; Carvalho, Lina; et al. "Characterization of a human Machado-Joseph disease neuronal cell model derived from induced pluripotent stem cells.". Paper presented in 12th International Society for Stem Cell Research (lSSCR 2015) Annual Meeting. Stockholm, Sweden., 2015.
  37. Sara Lopes (D715-EE08-5DD9); Rui Nobre; Clévio Nobrega; Neville Sanjana; Le Cong; Patrick Hsu; Fei Ann Ran; Feng Zhang; Pereira de Almeida, Luis. "Gene editing strategies towards Machado Joseph disease.". Paper presented in EMBO workshop: Modern DNA concepts and tools for safe gene transfer and modification. Institut de Biologie Génétique et Bio-Informatique (IBGBI), Evry-Val d'Essonne, France., 2015.
Journal article
  1. Miguel M. Lopes; Lopes, Sara M. ; Rafael Baganha; Carina Henriques; Ana C. Silva; Diana D. Lobo; Luísa Cortes; Luís Pereira de Almeida; Rui Jorge Nobre. "Isolation of Adeno-associated Viral Vectors Through a Single-step and Semi-automated Heparin Affinity Chromatography Protocol". Journal of Visualized Experiments (JoVE) 206 (2024): e66550.
    Published
  2. Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes; Pereira de Almeida, Luis ; Luísa Cortes; Rui Jorge Nobre. "A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification". Gene Therapy (2022): https://doi.org/10.1038/s41434-022-00372-z.
    10.1038/s41434-022-00372-z
  3. Vasconcelos-Ferreira, Ana; Martins, Inês Morgado; Lobo, Diana; Pereira, Dina; Miguel M. Lopes; Faro, Rosário; Lopes, Sara M. ; et al. "ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease". Molecular Therapy 30 1 (2022): 370-387. http://dx.doi.org/10.1016/j.ymthe.2021.07.012.
    Published • 10.1016/j.ymthe.2021.07.012
  4. Nobre, Rui Jorge; Lobo, Diana D.; Henriques, Carina; Duarte, Sonia P.; Lopes, Sara M. ; Silva, Ana C.; Miguel M. Lopes; et al. "MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3". Nucleic Acid Therapeutics (2021): http://dx.doi.org/10.1089/nat.2021.0020.
    Published • 10.1089/nat.2021.0020
  5. Lopes, Sara Monteiro; Faro, Rosário; Miguel M. Lopes; Onofre, Isabel; Mendonça, Nuno; Ribeiro, Joana; Januário, Cristina; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Protocol for the Characterization of the CAG Tract and Flanking Polymorphisms in Machado-Joseph Disease: Impact on Diagnosis and Development of Gene-Based Therapies". The Journal of Molecular Diagnostics (2020): http://dx.doi.org/10.1016/j.jmoldx.2020.03.003.
    10.1016/j.jmoldx.2020.03.003
  6. Silva, Ana C; Lobo, Diana D; Martins, Inês M; Lopes, Sara Monteiro; Henriques, Carina; Duarte, Sónia P; Dodart, Jean-Cosme; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders". Brain 143 2 (2020): 407-429. http://dx.doi.org/10.1093/brain/awz328.
    Published • 10.1093/brain/awz328
  7. Onofre, Isabel; Mendonça, Nuno; Lopes, Sara; Nobre, Rui; de Melo, Joana Barbosa; Carreira, Isabel Marques; Januário, Cristina; Gonçalves, António Freire; Pereira de Almeida, Luis. "Fibroblasts of Machado Joseph Disease patients reveal autophagy impairment". Scientific Reports 6 1 (2016): 28220. http://dx.doi.org/10.1038/srep28220.
    Published • 10.1038/srep28220
Newspaper article
  1. João Ramalho-Santos; Sara Varela Amaral; Marta Quatorze; João Cardoso; Luís Pereira de Almeida; André Caetano; Ana Catarina Vinhas; et al. "Uma Viagem Rara", Público, 2020, https://www.publico.pt/2020/02/28/ciencia/fotogaleria/viagem-rara-400481.
Thesis / Dissertation
  1. "Gene editing technologies to Machado-Joseph disease". PhD, Universidade de Coimbra Instituto de Investigação Interdisciplinar, 2021. https://estudogeral.uc.pt/handle/10316/105165.

Artistic / Performance

Theatre play
  1. Marionet theatre company. 2016. O Ponto que nos une.

Intellectual property

Provisional application for patent
  1. 2020. "Oligonucleotide primers for the characterization of the CAG tract and measurement of mutant mRNA in the context of polyglutamine disorders".
Activities

Oral presentation

Presentation title Event name
Host (Event location)
2024/02/09 Targeted silencing via AAV9 polycistronic microRNA therapy rescues SCA3 molecular, neuropathological and motor deficits 54th Meeting of the Portuguese Society of Pharmacology (SPF2024)
(Lisbon, Portugal)
2024/02 Mesenchymal Stromal Cell Therapeutic Potential to Restore Neurovascular Integrity in Spinocerebellar Ataxia Type 3 54th Meeting of the Portuguese Society of Pharmacology (SPF2024)
(Lisbon, Portugal)
2023/06/15 Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease 2023 CAG Triplet Repeat Disorder -Gordon Research Conference
(West Dover, Vermont, United States)
2023/02 Investigating the clock components and circadian activity in the YAC-MJD mouse model Meeting of the Portuguese Society of Pharmacology (SPF2023)
(Coimbra, Portugal)
2023/02 Extracellular vesicles delivery of CRISPR-Cas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3 54th Meeting of the Portuguese Society of Pharmacology (SPF2024)
(Lisbon, Portugal)
2022/06/27 Engineered nucleases targeting the ATXN3 gene for the therapy of Machado-Joseph disease (Flash Talk Presentation) 27th European Society for Animal Cell Technology Meeting (ESACT2022)
(Lisbon, Portugal)
2022/02/10 Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems for therapeutic genome editing Meeting of the Portuguese Society of Pharmacology (SPF2022)
(Porto, Portugal)
2022/02/10 Correction of ATXN3-CAG expansion in MJD patient-derived IPSC by CRISPR/Cas9 homology directed repair Meeting of the Portuguese Society of Pharmacology (SPF2022)
(Porto, Portugal)
2022 Modelling Machado-Joseph Disease in patient-derived isogenic lines by CRISPR/Cas9 homology directed repair 26th Annual Portuguese Society of Human Genetics Meeting (SPGH2022)
(Coimbra, Portugal)
2021/02/19 Phenotypic characterization of a relevant model of Machado-Joseph disease: The YAC-MJD mouse model. Meeting of the Portuguese Society of Pharmacology (SPF2021)
(Virtual Meeting, Portugal)
2021/02/19 Phenotypic characterization of neuronal cultures derived from Machado-Joseph disease induced pluripotent stem cells. Meeting of the Portuguese Society of Pharmacology (SPF2021)
(Virtual Meeting, Portugal)
2021/02/18 Allele-specific silencing of Machado-Joseph disease using antisense oligonucleotide therapeutics Meeting of the Portuguese Society of Pharmacology (SPF2021)
(Virtual Meeting, Portugal)
2020/11/25 Whole-Brain Biodistribution Analysis of Adeno-Associated Virus (AAV) with Single-Cell Resolution by Tissue Clearing and Light-Sheet Microscopy Early Career European Microscopy Congress 2020
(Virtual Meeting, United Kingdom)
2020/11/20 Permanent Inactivation of the ATXN3 Gene: A Possible Therapeutic Approach for Machado-Joseph Disease 24th Annual meeting of the Portuguese Society of Human Genetics (SPGH 2020)
(Virtual meeting, Portugal)
2020/02/07 CRISPR-mediated silencing of mutant ataxin-3 ameliorates motor symptoms of a Machado-Joseph disease model in vivo. XLX Meeting of the Portuguese Society of Pharmacology (SPF2020)
(Coimbra, Portugal)
2019/11/15 Reinstating levels of dysregulated autophagy-associated transcripts alleviates neuropathology and motor impairments of mouse models of Machado-Joseph Disease. International Ataxia Research Conference (IARC 2019).
(Washington, DC. , United States)
2019/11/14 CRISPR-Cas9 systems targeting ATXN3 as therapeutic approaches for Machado-Joseph Disease. Flash Talk Presentation. International Ataxia Research Conference (IARC 2019).
(Washington, DC, United States)
2019/11/13 CRISPR-Cas9 systems targeting ATXN3 as therapeutic approaches for Machado-Joseph Disease. 2019 International Machado-Joseph Research Disease Conference.
(Washington, DC., United States)
2019/11/12 Reinstating levels of dysregulated autophagy-associated transcripts alleviates neuropathology and motor impairments of mouse models of Machado-Joseph Disease. 2019 International Machado-Joseph Research Disease Conference.
(Washington, DC., United States)
2019/11/07 Whole-Brain Analysis of Adeno-Associated Virus (AAV) Biodistribution with Single-Cell Resolution by Tissue Clearing and Light-Sheet Microscopy. 2019 Spanish & Portuguese Advanced Optical Microscopy Meeting (SPAOM 2019).
(Coimbra, Portugal)
2019/11/07 A New Clearing Method for Whole-Brain Imaging with Single-Cell Resolution. 2019 Spanish & Portuguese Advanced Optical Microscopy Meeting 2019 (SPAOM 2019).
(Coimbra, Portugal)
2019/10/09 Whole-Brain Analysis of Adeno-Associated Virus (AAV) Biodistribution with Single-Cell Resolution by Tissue Clearing and Light-Sheet Microscopy. Flash Talk Presentation. EMBO | EMBL Symposium: Seeing Is Believing – Imaging the Molecular Processes of Life.
(Heidelberg, Germany)
2019/05/31 Permanent inactivation of the ATXN3 gene: A possible therapeutic approach for Machado-Joseph disease. XVI Meeting of the Portuguese Society for Neuroscience (SPN 2019).
(Lisbon, Portugal)
2019/05/22 CRISPR interference-mediated silencing of mutant ATXN3 decreases motor impairments of an in vivo Machado-Joseph disease model. 53rd Annual Scientific Meeting of the European Society for Clinical Investigation (ESCI).
(Coimbra, Portugal)
2018/05 Non-invasive brain delivery of hybrid extracellular vesicles for therapy of Machado-Joseph Disease (MJD). Annual Meeting ISEV2018.
(Barcelona, Spain)
2018/03/03 Tailoring gene editing strategies for the treatment of Machado-Joseph disease. Portuguese Society of Movement Disorders (SPDMov 2018).
(Curia, Portugal)
2018/02/17 Hybrid vesicles: a promising therapeutic approach for MJD and other neurodegenerative diseases. 1st Meeting on Vesicular Biology.
(Coimbra, Portugal)
2018/02/07 Protocol for the characterization of CAG tract and flanking polymorphisms in polyQ disorders: impact on the diagnosis and in the development of gene-based therapies. XLVIII Portuguese Society of Pharmacology Annual Meeting (SPF 2018).
(Lisbon, Portugal)
2018/02/07 Generation of a Machado-Joseph disease isogenic control induced pluripotent stem cell line. XLVIII Portuguese Society of Pharmacology Annual Meeting (SPF 2018).
(Lisbon, Portugal)
2018/02/07 AAV-Based tools for the development of cellular and animal models of Machado-Joseph disease. XLVIII Portuguese Society of Pharmacology Annual Meeting (SPF 2018).
(Lisbon, Portugal)
2018/02/07 CRISPR-Cas9-mediated pre-transcriptional silencing of mutant ataxin-3. XLVIII Portuguese Society of Pharmacology Annual Meeting (SPF 2018).
(Lisbon, Portugal)
2018/02/06 Reinstating levels of dysregulated autophagy-associated transcripts alleviates neuropathology and motor impairments of mouse models of Machado-Joseph disease. XLVIII Portuguese Society of Pharmacology Annual Meeting (SPF 2018).
(Lisbon, Portugal)
2018/01/15 Exosomes: tiny vesicles with great potential for MJD treatment. XII Spanish-Portuguese Conference on Controlled Drug Delivery.
(Coimbra, Portugal)
2017/09/30 TALEN and CRISPR gene-editing for treatment of Machado-Joseph disease. 2nd International Ataxia Research Conference (IARC 2017).
(Pisa, Italy)
2017/02/03 Gene editing strategies for Machado-Joseph disease. XLVII Portuguese Society of Pharmacology Annual Meeting (SPF 2017).
(Coimbra, Portugal)
2017/02/03 Pre-transcriptional silencing of ataxin-3 using a CRISPR-Cas9-derived system. XLVII Portuguese Society of Pharmacology Annual Meeting (SPF 2017).
(Coimbra, Portugal)

Event organisation

Event name
Type of event (Role) 		Institution / Organization
2013/12/18 - 2013/12/20 XI Annual CNC Meeting (2013/12/18 - 2013/12/20)
Meeting (Member of the Organising Committee)
 Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2012/06/02 - 2012/06/02 1st BEB Symposium "Science Dreams, challenges arise" (2012/06/02 - 2012/06/02)
Symposium (Member of the Organising Committee)
 Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Event participation

Activity description
Type of event 		Event name
Institution / Organization
2020/04/22 - 2020/06/30 Bootcamp in the framework of the innovation programme in neuroscience - Building Tomorrow Together
Other
 Building Tomorrow Together
Roche Farmaceutica Quimica Lda, Portugal
2016/11/18 - 2016/11/19 1st International Symposium - Brain Without Borders, held in Coimbra, Portugal. This event offered the opportunity to promote discussion on fundamental and translational research in stem cell biology applied to the Central Nervous System. A group of leading researchers from all over the world presented and discussed their work, sharing recent findings on 2D and 3D in vitro neuronal models, stem cell transplantation, in vivo gene therapy and neural tissue engineering.
Symposium
 Brain Without Borders
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2016/06/27 - 2016/07/01 Interdisciplinary educational program adressing concepts on how to offer "support for the aging society and create knowledge to deliver innovation and business." The course was part of the EIT Health Campus programme
Other
 Entrepreneurial Introductory Translational Research (EITR)
Exzellenzcluster CECAD in der Universität zu Köln, Germany
2016/03/17 - 2016/03/20 10th World Congress on Controversies in Neurology (CONy), Lisbon, Portugal. This event offered the opportunity to promote discussion on the molecular mechanisms of Machado-Joseph disease (MJD) and associated clinical features, discussing key considerations related with the translation from pre-clinical studies to clinical trials.
Congress
 10th World Congress on Controversies in Neurology (CONy)
2014 - 2014 Laboratory animal science category B Course, under FELASA guidelines. Course focused on the training of scientists to manipulate laboratory animals.
Other
 Training in Laboratory Animal Science
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2012/10 - 2012/11/03 Bloodborne pathogen for researchers' course
Other
Broad Institute, United States
2012/10 - 2012/10/24 Managing hazardous waste course
Other
Broad Institute, United States

Jury of academic degree

Topic
Role 		Candidate name (Type of degree)
Institution / Organization
2022/09/21 Evaluation of patients with neurological diseases due to nucleotide repeat expansions
(Thesis) Main arguer
 Madalena Maria Petronilo Marques (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal
2021/12/14 Impacto dos Polimorfismos de Nucleótido Único em Genes Associados a Doenças de Poliglutaminas para o Desenvolvimento de Terapias Génicas
(Thesis) Main arguer
 Sara Alexandra Nascimento Costa e Pires Martins (Master)
Universidade de Coimbra Faculdade de Farmácia, Portugal

Association member

Society Organization name Role
2019/02 - Current Sociedade Portuguesa de Neurociências
2012/09/20 - Current Ordem dos Farmacêuticos

Course / Discipline taught

Academic session Degree Subject (Type) Institution / Organization
2023/04/14 - 2023/04/14 Gene repair and models of disease Master in Pharmaceutical Biotechnology (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2023/03/03 - 2023/03/03 Gene editing in brain diseases Master in Molecular and Translational Neuroscience (Mestrado) Universidade de Coimbra Faculdade de Medicina, Portugal
2022/04 - 2022/08 Invited Assistant Professor Biologia Molecular (Mestrado integrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2022/05/27 - 2022/05/27 Therapies based on gene editing: Potentialities and limitations Integrated Master's degree in Medicine, Interdisciplinary Seminar of Applied Molecular Biology and Fundamentals of Neuroscience (Mestrado integrado) Universidade dos Açores, Portugal
2022/05/12 - 2022/05/12 Therapies based on gene editing: Potentialities and limitations Interdisciplinary Seminar of Applied Molecular Biology and Fundamentals of Neuroscience (Mestrado integrado) Universidade de Coimbra Faculdade de Medicina, Portugal
2022/04/12 - 2022/04/12 Therapies based on gene editing: Potentialities and limitations Advanced Therapies Course, Doctoral Programme in Experimental Biology and Biomedicine (Doutoramento) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2021/04/21 - 2021/04/21 Therapies based on gene editing: Potentialities and limitations Advanced Therapies course, Doctoral Programme in Experimental Biology and Biomedicine (Doutoramento) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2021/03/16 - 2021/03/16 Gene Editing Genética Molecular Humana, Mestrado Análises Clínicas (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2021/03/15 - 2021/03/15 Genotyping, Haplotypes and CAGs Genética Molecular Humana, Mestrado Análises Clínicas (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2020/05/02 - 2020/05/02 Gene repair and models of disease Terapêutica Molecular e Engenharia de Tecidos, Mestrado de Biotecnologia Farmacêutica (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2019/04/22 - 2019/04/22 Gene editing strategies in gene therapy Advanced Therapies course, Doctoral Programme in Experimental Biology and Biomedicine (Doutoramento) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2019/03/30 - 2019/03/30 Gene repair and models of disease Terapêutica Molecular e Engenharia de Tecidos, Mestrado de Biotecnologia Farmacêutica (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2018/05/08 - 2018/05/08 Nuclease-based genome editing technologies Advanced Therapies course, : Doctoral Programme in Experimental Biology and Biomedicine (Doutoramento) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2018/04/14 - 2018/04/14 Gene repair and models of disease Terapêutica Molecular e Engenharia de Tecidos, Mestrado de Biotecnologia Farmacêutica (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2017/06/16 - 2017/06/16 Therapies based on gene editing using CRISPR and TALEN: Potentialities and limitations Mestrado de Farmacologia Aplicada (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2017/03/25 - 2017/03/25 Gene repair and disease models Terapêutica Molecular e Engenharia de Tecidos, Mestrado de Biotecnologia Farmacêutica (Mestrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2017/03/16 - 2017/03/16 Aula Prática de Tecnologia Farmacêutica I Tecnologia Farmacêutica I (Mestrado integrado) Universidade de Coimbra Faculdade de Farmácia, Portugal
2016/06/25 - 2016/06/25 Gene edition: TALENs and CRISPR Mestrado de Farmacologia Aplicada Universidade de Coimbra Faculdade de Farmácia, Portugal
2016/06/16 - 2016/06/16 Gene editing strategies in gene therapy Advanced Therapies course, Doctoral Programme in Experimental Biology and Biomedicine (Doutoramento) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Mentoring / Tutoring

Topic Student name
2022/12 - 2023/06 MJDedit - Systems of gene editing for the ATXN3 gene Maria Adelaide Catarino Casquinha
2020/12 - 2022/08 Research fellow in the context of a project funded by a Pharmaceutical company Ana Daniela Oliveira
2019/09 - 2020/12 Phenotypic characterization of cellular and animal models of Machado-Joseph disease Ana Daniela Oliveira
Distinctions

Award

2020 First prize of the innovation programme in neuroscience: Building Tomorrow Together (Roche initiative) with the project ExoTreat
Roche Farmaceutica Quimica Lda, Portugal
2020 Best Basic Research award for the work presented at the Portuguese Society of Human Genetics
Sociedade Portuguesa de Genética Humana (SPGH), Portugal
2018 Best work in basic science presented at the Portuguese Society of Movement Disorders
Sociedade Portuguesa das doenças do Movimento (SPDMov), Portugal
2017 EIT Health PhD transition Fellowship (3rd prize) for the project "ExoEditor - A combined therapeutic strategy for Machado-Joseph disease"
EIT Health, Germany

Other distinction

2019 Travel bursary to attend the MJD Satellite Conference in Washington DC, USA.
MJD Foundation, Australia
2017 Young Investigator Bursary to attend the IARC2017 congress
Ataxia UK, United Kingdom
2016 Travel Award for the CECAD Short Course on Entrepreneurial Introductory Translational Research (EITR)
Exzellenzcluster CECAD in der Universität zu Köln, Germany
2011 Individual 4-year PhD Fellowship (Ref. SFRH/BD/51673/2011).
Associação para a Inovação e Desenvolvimento da FCT, Portugal