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Miguel Lopes holds a BSc in Biochemistry (2015) and an MSc in Biomedical Research (2017, graded 19/20) from the University of Coimbra. His master’s thesis, developed at the Vectors and Gene Therapy group (CNC-UC), focused on generating novel cellular and animal models of SCA3/MJD using mosaic rAAV vectors, receiving a top mark (20/20) and the BEB/Bluepharma Award (2018). Between 2017 and 2019, he worked as a research fellow on the ImaGene project, including a research stay at Carl Zeiss AG (Germany), and presented his work through flash talks, posters, oral communications and an invited workshop lecture. He subsequently contributed to the BrainHealth 2020 project as a research fellow and has been actively collaborating with ViraVector@UC on rAAV purification protocols. He is author/co-author of 9 peer-reviewed publications and 3 preprints covering topics of rAAV production, purification and biodistribution, as well as disease modelling, characterisation and gene therapy applications in the context of SCA3/MJD. One of his publications was selected as Editor’s Choice and featured on the front cover of the Gene Therapy Journal. He has contributed to the development of different projects, being author/co-author of 32 oral communications and 35 posters, and actively participates in congresses, workshops and training activities related to research development, innovation and translation of research findings into clinical applications. Through his research and training, he developed expertise in cell culture techniques, molecular biology, fast protein liquid chromatography, luminescence assays, immunocytochemistry, immunohistochemistry, tissue clearing, 3D fluorescence imaging, microscopy image analysis, animal experimentation (certified by DGAV), among others. Since December 2020, he has been pursuing a PhD in Experimental Biology and Biomedicine at CNC/IIIUC, funded by an FCT fellowship, to generate new models of polyglutamine diseases and therapeutic strategies, using rAAVs.
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Identificação

Identificação pessoal

Nome completo
Miguel Monteiro Lopes
Género
Masculino

Nomes de citação

  • Miguel M. Lopes
  • Lopes, M. M.
  • Miguel Lopes

Identificadores de autor

Ciência ID
8A16-C433-C6FF
ORCID iD
0000-0001-9869-0203

Telefones

Telefone
  • 239820190 (Profissional)

Domínios de atuação

  • Ciências Médicas e da Saúde - Biotecnologia Médica - Diagnóstico e Terapias de Base Genética
  • Ciências Médicas e da Saúde - Biotecnologia Médica - Biotecnologia Aplicada à Saúde
  • Ciências Médicas e da Saúde - Biotecnologia Médica - Tecnologias que envolvem a Manipulação de Células,Tecidos,Órgãos ou todo o Organismo
  • Ciências Médicas e da Saúde - Medicina Básica - Neurociências
  • Ciências Médicas e da Saúde - Medicina Básica - Genética Humana
  • Ciências Naturais - Ciências Biológicas - Bioquímica
  • Ciências Naturais - Ciências Biológicas - Biologia Molecular
  • Ciências Naturais - Ciências Biológicas - Biologia Celular
  • Ciências Naturais - Ciências Biológicas - Microbiologia

Idiomas

Idioma Conversação Leitura Escrita Compreensão Peer-review
Português (Idioma materno)
Inglês Utilizador proficiente (C1) Utilizador proficiente (C2) Utilizador proficiente (C2) Utilizador proficiente (C2)
Espanhol; Castelhano Utilizador elementar (A1) Utilizador independente (B1) Utilizador elementar (A1) Utilizador independente (B1)
Formação
Grau Classificação
2020/12/09 - 2025
Em curso
 Doutoramento em Biologia Experimental e Biomedicina (PDBEB) (Doutoramento)
Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal
2020/03/19 - 2020/06/29
Concluído
 Curso de Formação de Análise Estatística e Multivariada em R: aplicações em sistemas com vários componentes (Outros)
Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
 20
2020/02/18 - 2020/05/25
Concluído
 Curso de Formação de Inglês para Fins Académicos II (nível intermédio/avançado) (Outros)
Universidade de Coimbra Faculdade de Letras, Portugal
2020/01/02
Concluído
 Autorização/certificação para realizar procedimentos em animais (Pequenos roedores e Peixes) (Outros)
Direção Geral de Alimentação e Veterinária (DGAV), Portugal
2019/01/04 - 2019/01/24
Concluído
 Curso Pós-Graduado em Experimentação Animal (Prático), para as funções A e D de Roedores e Peixes (Pós-Graduação)
Sociedade Portuguesa de Ciências em Animais de Laboratório - SPCAL, Portugal

Universidade Nova de Lisboa Faculdade de Ciências Médicas, Portugal
19
2015/08/05 - 2017/09/12
Concluído
 Investigação Biomédica (Mestrado)
Especialização em Neurobiologia
Universidade de Coimbra Faculdade de Medicina, Portugal
"AAV-Based Tools for the Development of Cellular and Animal Models of Machado-Joseph Disease" (TESE/DISSERTAÇÃO)
 19
2017/02/23
Concluído
 Curso Teórico de 20h de Ciências de Animais de Laboratório Desenhado Segundo as Recomendações da FELASA para a Categoria B (Outros)
Sociedade Portuguesa de Ciências em Animais de Laboratório - SPCAL, Portugal

Universidade de Coimbra Faculdade de Farmácia, Portugal
83%
2011/09/19 - 2015/01/30
Concluído
 Bioquímica (Licenciatura)
Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
 14.35
Percurso profissional

Ciência

Categoria Profissional
Instituição de acolhimento 		Empregador
2020/05/04 - 2021/12/31 Investigador (Investigação) Universidade de Coimbra, Portugal
Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal
2017/11/01 - 2019/12/31 Investigador (Investigação) Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2015/03/01 - 2016/08/01 Estagiário de Investigação (Investigação) Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2014/03/01 - 2014/07/01 Estagiário de Investigação (Investigação) Laboratório de Micobacteriologia Molecular e Microbioma, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2013/07/01 - 2013/09/01 Estagiário de Investigação (Investigação) Laboratório de Microbiologia - Departamento de Bioquímica da Faculdade de Ciências e Tecnologia da Universidade de Coimbra, Portugal
Laboratório de Microbiologia - Departamento de Bioquímica da Faculdade de Ciências e Tecnologia da Universidade de Coimbra, Portugal
Projetos

Bolsa

Designação Financiadores
2022/01/01 - 2025/12/31 Enhanced AAV vector toolkit for the generation of novel animal models and therapeutic strategies for polyglutamine diseases
2021.05776.BD
Bolseiro de Doutoramento
Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal

ViraVector - Centro de Neurociências e Biologia Celular da Universidade de Coimbra (CNC-UC), Portugal

Centre for Innovative Biomedicine and Biotechnology (CIBB), Portugal

Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal
Fundação para a Ciência e a Tecnologia
Em curso

Projeto

Designação Financiadores
2022/07/01 - 2023/06/30 ModelPolyQ 2.0 – Advanced Animal Models for Polyglutamine Diseases 2.0
CENTRO-01-0145-FEDER-181258
Investigador
Universidade de Coimbra, Portugal
 Programa Operacional Regional do Centro (CENTRO2020)
Concluído
2022/01/01 - 2023/06/30 Genome editing to rescue Machado-Joseph disease by alternative splicing
EXPL/MED-NEU/0936/2021
Investigador
Universidade de Coimbra, Portugal

Universidade de Coimbra Instituto de Investigação Clínica e Biomédica de Coimbra, Portugal
Fundação para a Ciência e a Tecnologia
Concluído
2020/04/24 - 2021/12/31 BrainHealth 2020 - Detecção Precoce, Neuro-modulação e Terapias Avançadas para Neuropatologias
CENTRO-01-0145-FEDER-000008
Bolseiro de Investigação
CNC.IBILI, Portugal
 Programa Operacional Regional do Centro (CENTRO2020)

Fundo Europeu de Desenvolvimento Regional (FEDER)
Concluído
2017/11/01 - 2019/12/31 Visualização da Terapia Génica do Sistema Nervoso Central; "Imaging Gene Therapy in the Brain - ImaGene"
PTDC/BBB-NAN/0932/2014
Bolseiro de Investigação
Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal
 Fundação para a Ciência e a Tecnologia
Concluído

Outro

Designação Financiadores
2020/01/01 - Atual Centro de Inovação em Biomedicina e Biotecnologia
UIDB/04539/2020
UIDP/04539/2020
Investigador
Centre for Innovative Biomedicine and Biotechnology (CIBB), Portugal
 Fundação para a Ciência e a Tecnologia
Em curso
2017/03/01 - Atual ViraVector - Viral Vector for Gene Transfer Core Facility
CENTRO-01-0145-FEDER-022095
Investigador
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Em curso
2020/06/01 - 2020/11/30 Research4COVID-19 - Validação expedita e Fabrico em larga escala de um dispositivo de sopro para colheita com inativação de amostras para deteção do SARS-CoV-2
Research4COVID-19 nº 269_596884125
Investigador
Universidade de Coimbra, Portugal

ViraVector - Centro de Neurociências e Biologia Celular da Universidade de Coimbra (CNC-UC), Portugal

Institute of Interdisciplinary Research, Portugal
Fundação para a Ciência e a Tecnologia
Concluído
2020/05/04 - 2020/06/04 Covid-19 Lab at CNC / ViraVector
N.A.
Investigador
Concluído
2015/03/01 - 2019/12/31 CNC. IBILI
UID/NEU/04539/2013
UID/NEU/04539/2019
Investigador
CNC.IBILI, Portugal
Concluído
Produções

Publicações

Artigo em jornal
  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.
Artigo em revista
  1. Silva, Ana Carolina; Lopes, Miguel Monteiro; Lobo, Diana Duarte; Henriques, Carina; Pereira de Almeida, Luís; Nobre, Rui Jorge. "Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics". Trends in Molecular Medicine 31 11 (2025): 1065-1066. https://doi.org/10.1016/j.molmed.2025.06.007.
    Publicado • 10.1016/j.molmed.2025.06.007
  2. Henriques, Carina; Silva, Marta; Silva, António; Rufino-Ramos, David; Lopes, Miguel Monteiro; Badin, Romina Aron; Hantraye, Philippe; Pereira de Almeida, Luís; Nobre, Rui Jorge. "Differential impact of mutant Ataxin-3 in hindbrain regions: further evidence of white matter loss as a core pathological feature". Experimental Neurology 393 (2025): 115413. https://doi.org/10.1016/j.expneurol.2025.115413.
    Publicado • 10.1016/j.expneurol.2025.115413
  3. Henriques, Carina; Albuquerque, Patrícia; Rufino-Ramos, David; Lopes, Miguel M.; Leandro, Kevin; Miranda, Catarina O.; Almeida, Rita; et al. "Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease". Molecular Therapy (2025): https://doi.org/10.1016/j.ymthe.2025.10.022.
    10.1016/j.ymthe.2025.10.022
  4. Lopes, Miguel M.; Lopes, Sara M.; Baganha, Rafael; Henriques, Carina; Silva, Ana C.; Lobo, Diana D.; Cortes, Luisa; Pereira de Almeida, Luis ; Nobre, Rui Jorge. "Isolation of Adeno-associated Viral Vectors Through a Single-step and Semi-automated Heparin Affinity Chromatography Protocol". Journal of Visualized Experiments 206 (2024): https://doi.org/10.3791/66550.
    Acesso aberto • Publicado • 10.3791/66550
  5. Henriques, Carina; Lopes, Miguel M; Silva, Ana C; Lobo, Diana D; Badin, Romina Aron; Hantraye, Philippe; Pereira de Almeida, Luís; Nobre, Rui Jorge. "Viral-based animal models in polyglutamine disorders". Brain 147 4 (2024): 1166-1189. http://dx.doi.org/10.1093/brain/awae012.
    Publicado • 10.1093/brain/awae012
  6. Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes; Luís Pereira de Almeida; 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
  7. Rui Jorge Nobre; Diana D. Lobo; Carina Henriques; Sonia P. Duarte; Sara M. Lopes; Ana C. Silva; 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): https://doi.org/10.1089/nat.2021.0020.
    10.1089/nat.2021.0020
  8. Vasconcelos-Ferreira, Ana; Martins, Inês Morgado; Lobo, Diana; Pereira, Dina; Lopes, Miguel M.; Faro, Rosário; Lopes, Sara M.; et al. "ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease". Molecular Therapy (2021): http://dx.doi.org/10.1016/j.ymthe.2021.07.012.
    No prelo • 10.1016/j.ymthe.2021.07.012
  9. Sara Monteiro Lopes; Rosário Faro; Miguel Monteiro Lopes; Isabel Onofre; Nuno Mendonça; Joana Ribeiro; Cristina Januário; Rui Jorge Nobre; Luís Pereira de Almeida. "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): https://doi.org/10.1016/j.jmoldx.2020.03.003.
    10.1016/j.jmoldx.2020.03.003
Poster em conferência
  1. Pena, Frederico; Fernandes, Ana Rita; Miguel M. Lopes; Núñez, Jisette; Faro, Rosario; Pires, M.; Pereira, A.; et al. "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". Trabalho apresentado em ESGCT/SETGYC Collaborative Congress, 2025.
  2. Miguel M. Lopes; Lopes, Sara M. ; R Baganha; Henriques, Carina; Silva, Ana Carolina; Lobo, Diana Duarte; Faro, Rosario; et al. Autor correspondente: Nobre, Rui. "Streamlined heparin affinity chromatography enables efficient rAAV1/2 purification with superior CNS transduction compared to rAAV9 upon neonatal ICM delivery". Trabalho apresentado em ESGCT/SETGYC Collaborative Congress, 2025.
  3. 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". Trabalho apresentado em ESGCT/SETGYC Collaborative Congress, 2025.
  4. Lopes, Sara M.; Miguel M. Lopes; Santana, Magda M; Oliveira, Daniela; Fernandes, Ana Rita; Casquinha, Maria; Vasconcelos-Ferreira, Ana; et al. "CRISPR-Cas9-Mediated ATXN3 Gene Inactivation as a Promising Gene Therapy for Machado-Joseph Disease". Trabalho apresentado em 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), 2025.
  5. Lopes, Sara M.; Miguel M. Lopes; Ana Daniela Oliveira; Magda M. Santana; Ana R. Fernandes; Ana Vasconcelos-Ferreira; Pereira, Dina; et al. "Therapeutic Potential of CRISPR-Cas9-Mediated ATXN3 Gene Inactivation in Machado-Joseph Disease". Trabalho apresentado em Rare Disease Research Conference 2025, 2025.
  6. Henriques, Carina; Silva, Marta; da Silva, António; Rufino-Ramos, David; Lopes, M. M.; Aron Badin, Romina; Hantraye, Philippe; Pereira de Almeida, L; Nobre, Rui Jorge. "Novel evidence of white matter contribution in Spinocerebellar Ataxia type 3 pathology". Trabalho apresentado em International Congress for Ataxia Research (ICAR) 2024, 2024.
  7. Gabriel, Guilherme L.; Silva, Marta; Batista, Alexandre; Leandro, Kevin; Almeida, Rita; Silva, Ana Carolina; Lopes, M. M.; et al. "Hacking Biology: Optimizing AAV-based gene delivery for enhanced region-specific brain targeting". Trabalho apresentado em 31st Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2024.
  8. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Lopes, M. M.; Leandro, Kevin; Pereira, Dina; et al. "Intra-cisterna magna administration of allele-specific AAV-based therapy alleviates neuropathology and motor deficits in spinocerebellar ataxia type 3". Trabalho apresentado em 31st Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2024.
  9. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Miguel M. Lopes; Leandro, Kevin; Pereira, Dina; et al. "Allele-specific silencing for Spinocerebellar Ataxia Type 3 via AAV9 polycistronic microRNA-based therapy". Trabalho apresentado em Annual Meeting of the Doctoral Program in Experimental Biology and Biomedicine (PDBEB day), 2024.
  10. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Lopes, M. M.; Pereira, Dina; Duarte, S.P.; et al. "Allele-specific silencing of mutant ATXN3 mitigates motor deficits and neuropathology in a severely impaired mouse model of spinocerebellar ataxia type 3 (SCA3)". Trabalho apresentado em American Society of Gene Therapy (ASGCT) 27th Annual Meeting, 2024.
  11. S. M. Lopes; Miguel M. Lopes; R. J. Nobre; A. V. Ferreira; D. Pereira; A. R. Fernandes; I. Barros; et al. "Targeting the ATXN3 gene with CRISPR-Cas9 for the therapy of Machado-Joseph disease". Trabalho apresentado em 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  12. A. C. Silva; C. Henriques; D. D. Lobo; D. Pereira; S. P. Duarte; S. M. Lopes; 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)". Trabalho apresentado em 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  13. C. Henriques; I. Barros; A. Silva; Miguel M. Lopes; R. A. Badin; P. Hantraye; L. Pereira de Almeida; R. J. Nobre. "Generation of lentiviral-based mouse models of spinocerebellar ataxias: where to target?". Trabalho apresentado em 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  14. A. C. Silva; C. Henriques; D. D. Lobo; A. R. Fernandes; Miguel M. Lopes; D. Pereira; S. P. Duarte; 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". Trabalho apresentado em 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  15. Henriques, Carina; Miguel M. Lopes; Albuquerque, Patrícia; Rufino-Ramos, David; Gaspar, Laetitia S; Lobo, Diana Duarte; Silva, Ana Carolina; et al. "Isolation of Natural and Biologically Active EV-AAVs for Brain Gene Delivery". Trabalho apresentado em American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, 2023.
  16. Pedro Perdigão; Miguel Dias; Sara Lopes; 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". Trabalho apresentado em American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, 2023.
  17. 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.". Trabalho apresentado em 26th Annual Portuguese Society of Human Genetics Meeting, 2022.
  18. C. Henriques; Miguel M. Lopes; P. Albuquerque; D. Rufino-Ramos; L. Gaspar; D. Lobo; A. C. Silva; et al. "Production and isolation of natural and biologically active exo-AAvs for brain gene delivery". Trabalho apresentado em ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, 2022.
  19. P. Perdigão; C. Matos; S. Lopes; R. Fernandes; Miguel M. Lopes; A. Conceição; R. Faro; et al. "Modelling Machado-Joseph Disease in patient-derived isogenic lines by CRISPR/Cas9-mediated correction of ATXN3-CAG expansion". Trabalho apresentado em ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, 2022.
  20. Miguel M. Lopes; J. Paysan; J. Rino; S. M. Lopes; 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". Trabalho apresentado em ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, 2022.
  21. Sara M. Lopes; Miguel M. Lopes; Rui J. Nobre; Clévio Nóbrega; Carlos A. Matos; Ana Vasconcelos-Ferreira; Dina Pereira; et al. "Engineered nucleases targeting the ATXN3 gene for the therapy of Machado-Joseph disease". Trabalho apresentado em 27th European Society for Animal Cell Technology Meeting (ESACT2022), 2022.
  22. Rafael Baganha; Miguel M. Lopes; Carina Henriques; David Rufino-Ramos; Rui Jorge Nobre; Luís Pereira de Almeida. "Affinity chromatography-based protocols for purification of neurotropic AAV vectors". Trabalho apresentado em 27th European Society for Animal Cell Technology Meeting (ESACT2022), 2022.
  23. Pedro Perdigão; Carlos Matos; Sara Lopes; Rita Fernandes; Miguel M. Lopes; Rosário Faro; Magda Santana; Rui Nobre; Luís Pereira de Almeida. "Precise and scarless correction of ATXN3-CAG expansion by CRISPR/Cas9 for the generation of patient-derived MJD isogenic iPSC". Trabalho apresentado em XVII Meeting of the Portuguese Society for Neuroscience, 2021.
  24. Rafael Baganha; Miguel M. Lopes; Carina Henriques; David Rufino-Ramos; Luís Pereira de Almeida; Rui Jorge Nobre. "Development of Affinity-Based Purification Protocols for AAV Vectors targeting the Central Nervous System". Trabalho apresentado em XVII Meeting of the Portuguese Society for Neuroscience - SPN 2021, 2021.
  25. Inês T. Afonso; Ana Carolina Silva; Carina Henriques; Miguel M. Lopes; Rafael Baganha; Patrícia Lima; Rebekah Kopenol; et al. "Brain distribution, spreading and tropism of a recently developed gene delivery system - mixCapAAV1/2vector". Trabalho apresentado em AIMS Meeting: Annual International (bio)Medical Students Meeting, 2021.
  26. Henriques C.; Lopes M. M.; Albuquerque P. R.; Rufino-Ramos D.; Gaspar L.; Lobo D.; Duarte S.; et al. "Extracellular Vesicles-Associated AAVs as New Gene Delivery Vectors for the Treatment of Brain Disorders". Trabalho apresentado em 3rd BEB Day, Winner of the BEB/BluePharma Award, 3rd Edition, 2019.
  27. Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes; 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". Trabalho apresentado em 1st Annual Meeting in Pharmaceutical Sciences, 2019.
  28. Sara Lopes; Carlos Matos; André Conceição; Frederico Pena; Miguel Lopes; Rui Nobre; Clévio Nóbrega; Ana Ferreira; Feng Zhang. "CRISPR-Cas9 Systems Targeting ATXN3 as Therapeutic Approaches for Machado-Joseph Disease". Trabalho apresentado em 2019 International Ataxia Research Conference (IARC 2019), 2019.
  29. Ana Ferreira; Inês Martins; Diana Lobo; Dina Pereira; Miguel Lopes; Sara Lopes; Rosário Faro; et al. "Reinstating Levels of Dysregulated Autophagy-Associated Transcripts Alleviates Neuropathology and Motor Impairments of Mouse Models of Machado-Joseph Disease". Trabalho apresentado em 2019 International Ataxia Research Conference (IARC 2019), 2019.
  30. Sara Lopes; Carlos Matos; André Conceição; Frederico Pena; Miguel Lopes; Rui Nobre; Clévio Nóbrega; et al. "CRISPR-Cas9 Systems Targeting ATXN3 as Therapeutic Approaches for Machado-Joseph Disease". Trabalho apresentado em 2019 International Machado-Joseph Research Disease Conference, 2019.
  31. Ana Ferreira; Inês Martins; Diana Lobo; Dina Pereira; Miguel Lopes; Sara Lopes; Rosário Faro; et al. "Reinstating Levels of Dysregulated Autophagy-Associated Transcripts Alleviates Neuropathology and Motor Impairments of Mouse Models of Machado-Joseph Disease". Trabalho apresentado em 2019 International Machado-Joseph Research Disease Conference, 2019.
  32. Henriques C.; Lopes M. M.; Albuquerque P. R.; Rufino-Ramos D.; Gaspar L.; Lobo D.; Duarte S.; et al. "Extracellular Vesicles-Associated AAVs as Gene Delivery Vectors for the Treatment of Brain Diseases". Trabalho apresentado em 5th GEIVEX Symposium, 2019.
  33. Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes; 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". Trabalho apresentado em EMBO | EMBL Symposium: Seeing Is Believing – Imaging the Molecular Processes of Life, 2019.
  34. Ana Vasconcelos-Ferreira; Inês Morgado Martins; Diana Lobo; Dina Pereira; Miguel Lopes; Sara Lopes; Rosário Faro; et al. "Reinstating Levels of Dysregulated Autophagy-Associated Transcripts Alleviates Neuropathology and Motor Impairments of Mouse Models of Machado-Joseph Disease". Trabalho apresentado em XVI Meeting of the Portuguese Society for Neuroscience (SPN), 2019.
  35. Lopes, S. M.; 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". Trabalho apresentado em 2nd International Ataxia Research Conference (IARC), 2017.
Pré-impressão
  1. Lopes, Sara Monteiro; Lopes, Miguel Monteiro; Oliveira, Daniela; Santana, Magda Matos; Fernandes, Ana Rita; Vasconcelos-Ferreira, Ana; Pereira, Dina; et al. "Gene Editing for ATXN3 Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced Toxicity". 2025. https://doi.org/10.1101/2025.02.14.637261.
    10.1101/2025.02.14.637261
  2. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana D.; Fernandes, Ana Rita; Lopes, Miguel M.; Leandro, Kevin; Pereira, Dina; et al. "rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3". 2025. https://doi.org/10.1101/2025.10.02.680011.
    10.1101/2025.10.02.680011
  3. Henriques, Carina; Lobo, Diana Duarte; Silva, Ana Carolina; Fernandes, Ana Rita; Lopes, Miguel Monteiro; Fayard, Audrey; Jan, Caroline; et al. "AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3". 2025. https://doi.org/10.1101/2025.10.02.680027.
    10.1101/2025.10.02.680027
Resumo em conferência
  1. No authors listed. "Proceedings of the 24th Annual Meeting of the Portuguese Society of Human Genetics (SPGH: Sociedade Portuguesa de Genética Humana)". Trabalho apresentado em Portuguese Society of Human Genetics (SPGH), Lisbon, 2021.
    Publicado • 10.1097/md.0000000000023585
  2. Lopes, Sara M. ; Miguel M. Lopes; Rui Nobre; Clévio Nóbrega; Matos, Carlos A; Ana Vasconcelos-Ferreira; Neville Sanjana; et al. Autor correspondente: Pereira de Almeida, Luis. "Permanent inactivation of the ATXN3 gene: a possible therapeutic approach for Machado-Joseph disease". Trabalho apresentado em 24th Annual Meeting of the Portuguese Society of Human Genetics (SPGH), Virtual Conference, 2021.
    Publicado
Tese / Dissertação
  1. Miguel M. Lopes. "AAV-Based Tools For The Development Of Cellular and Animal Models of Machado-Joseph Disease". Mestrado, Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, 2017.

Artística / Interpretação

Obra teatral
  1. Mário Montenegro; Sara Amaral; João Cardoso; Marta Quatorze; Miguel Lopes; Diana Lobo; Carina Henriques; et al. 2019. Holy CIBB.
Atividades

Apresentação oral de trabalho

Título da apresentação Nome do evento
Anfitrião (Local do evento)
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 29th Annual Meeting of the Portuguese Society of Human Genetics (SPGH)
(Coimbra, Portugal)
2024/11/13 CRISPR-Cas9-mediated ATXN3 gene inactivation as a potential therapeutic approach for Machado-Joseph disease 2024 International Congress for Ataxia Research (ICAR2024)
(London, Reino Unido)
2024/11/12 Allele-specific silencing of mutant ataxin-3 via single administration of AAV9 vectors mitigates neuropathology and motor deficits in Spinocerebellar Ataxia Type 3 2024 International Congress for Ataxia Research (ICAR2024)
(London, Reino Unido)
2024/02/07 Hacking biology: optimizing AAV-based gene delivery for enhanced brain region-specific targeting 54th Meeting of the Portuguese Society of Pharmacology (SPF2024)
(Lisbon, Portugal)
2024/02/07 Generation of lentiviral-based mouse models of spinocerebellar ataxias: where to target? 54th Meeting of the Portuguese Society of Pharmacology (SPF2024)
(Lisbon, Portugal)
2024/02/07 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)
2023/11/24 Polycistronic artificial microRNAs delivered by AAV9 alleviate Spinocerebellar Ataxia Type 3 (SCA3) Pharma PhD Day - Annual Meeting of PhD Students in Pharmaceutical Sciences
(Coimbra, Portugal)
2023/05/31 A novel pipeline for whole-brain biodistribution analysis of AAVs using tissue clearing and light-sheet fluorescence microscopy (Invited lecturer) Advanced Imaging Methods in Neuroscience, EIT Health Ageing PhD School
2023/05/16 Isolation of Natural and Biologically Active EV-AAVs for Brain Gene Delivery American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting
(Los Angeles, Estados Unidos)
2022/11/18 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)
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 Correction of ATXN3-CAG expansion in MJD patient-derived iPSC by CRISPR/Cas9 homology directed repair LII Meeting of the Portuguese Society of Pharmacology (SPF) / XL Clinical Pharmacology Meating / XXI Toxicology Meating
(Porto, Portugal)
2021/02/19 Development of Single-Step Affinity Chromatography Protocols for the Purification of Adeno-Associated Viral Vectors 51st Annual Meating of the Portuguese Society of Pharmacology (SPF) / 39th Clinical Pharmacology Meating / 20th Toxicology Meating
(Lisbon, Portugal)
2021/02/19 Phenotypic Characterization of a Relevant Model Of Machado-Joseph Disease: The YAC-MJD Mouse Model 51st Annual Meating of the Portuguese Society of Pharmacology (SPF) / 39th Clinical Pharmacology Meating / 20th Toxicology Meating
(Lisbon, Portugal)
2021/02/18 MixCapAAV1/2 Vector: A Novel Gene Delivery System Distribution, Spreading and Tropism Analysis in Normal Mice 51st Annual Meating of the Portuguese Society of Pharmacology (SPF) / 39th Clinical Pharmacology Meating / 20th Toxicology Meating
(Lisbon, Portugal)
2020/11/25 Whole-Brain Biodistribution Analysis of Adeno-Associated Virus (AAV) with Single-Cell Resolution by Tissue Clearing and Light-Sheet Microscopy Virtual Early Career European Microscopy Congress 2020
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
Portuguese Society of Human Genetics (Lisbon, Portugal)
2019/11/15 Extracellular Vesicles-Associated AAVs as Gene Delivery Vectors for the Treatment of Brain Disorders 1st Annual Meeting in Pharmaceutical Sciences
(Coimbra, Portugal)
2019/11/14 Reinstating Levels of Dysregulated Autophagy-Associated Transcripts Alleviates Neuropathology and Motor Impairments of Mouse Models of Machado-Joseph Disease 2019 International Ataxia Research Conference (IARC 2019)
(Washington DC., Estados Unidos)
2019/11/14 CRISPR-Cas9 Systems Targeting ATXN3 as Therapeutic Approaches for Machado-Joseph Disease. Flash Talk Presentation 2019 International Ataxia Research Conference (IARC 2019)
(Washington DC., Estados Unidos)
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., Estados Unidos)
2019/11/12 CRISPR-Cas9 Systems Targeting ATXN3 as Therapeutic Approaches for Machado-Joseph Disease 2019 International Machado-Joseph Research Disease Conference
(Washington DC., Estados Unidos)
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 (SPAOM 2019)
(COimbra, Portugal)
2019/11/07 Extracellular vesicles-associated AAVs as gene delivery vectors for the treatment of brain diseases 5th GEIVEX Symposium
(Granada, Espanha)
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, Alemanha)
2019/05/30 Permanent Inactivation of the ATXN3 Gene: A Possible Therapeutic Approach for Machado-Joseph Disease XVI Meeting of the Portuguese Society for Neuroscience (SPN)
(Lisboa, Portugal)
2019/02/06 Extracellular Vesicles-Associated AAVs as Gene Delivery Vectors for the Treatment of Brain Diseases XLIX Annual Meating of the Portuguese Society of Pharmacology (SPF) / XXXVII Clinical Pharmacology Meating / XVIII Toxicology Meating
(Porto, Portugal)
2018/03/02 Tailoring gene editing strategies for the treatment of Machado-Joseph disease Portuguese Society of Movement Disorders (SPDMov 2018)
(Curia, Portugal)
2018/02/07 AAV-Based Tools For The Development Of Cellular And Animal Models Of Machado-Joseph Disease XLVIII Annual Meating of the Portuguese Society of Pharmacology (SPF) / XXXVI Clinical Pharmacology Meating / XVII Toxicology Meating
(Lisboa, Portugal)
2018/02/06 Reinstating Levels of Dysregulated Autophagy-Associated Transcripts Alleviates Neuropathology and Motor Impairments of Mouse Models of Machado-Joseph Disease XLVIII Annual Meating of the Portuguese Society of Pharmacology (SPF) / XXXVI Clinical Pharmacology Meating / XVII Toxicology Meating
(Lisboa, Portugal)
2017/09/30 TALEN and CRISPR gene-editing for treatment of Machado-Joseph Disease 2nd International Ataxia Research Conference (IARC)
(Pisa, Itália)

Organização de evento

Nome do evento
Tipo de evento (Tipo de participação) 		Instituição / Organização
2025/06/24 - 2025/06/25 Participated as a lecturer and mentor in the theoretical and practical mini-course, “Viral Vectors: Production, Purification and Characterisation”, held at the ViraVector national research infrastructure at the University of Coimbra. The mini-course spanned a total of 10 h. It formed an integral part of the “Placements in Portugal” programme of the CAPACITY 2023 project and was delivered to six participants from the project's partner organisations: the National Drug Authority in Uganda and the Muhimbili University of Health Sciences in Tanzania. (2025/06/24 - 2025/06/25)
Oficina (workshop) (Outra)
 ViraVector - Centro de Neurociências e Biologia Celular da Universidade de Coimbra (CNC-UC), Portugal

Participação em evento

Descrição da atividade
Tipo de evento 		Nome do evento
Instituição / Organização
2023/03/28 - 2023/03/31 Attended the European Society for Gene and Cell Therapy (ESGCT) 6th Annual Spring School held in Giens, France, on March 28th-31th, 2023.
Oficina (workshop)
2022/11/08 - 2022/12/06 Successfully concluded the training programme in Bioentrepreneurship, BioSTART, organized by the Center for Neuroscience and Cell Biology of the University of Coimbra in consortium with Biocant and IATV that took place in Coimbra, Portugal from November 8th to December 6th, 2022.
Oficina (workshop)
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Biocant Associação de Transferência de Tecnologia, Portugal
2022/09/25 - 2022/09/29 Attended the second Bioprocessing and Manufacturing of Gene and Cell Therapy Products Course organized by the European Society for Animal Cell Technology that took place in Llafranc, Girona, Spain on September 25th - 29th, 2022.
Oficina (workshop)
2022/06/21 - 2022/06/21 Participation in the activity "Innocore challenges: students' and companies' perspectives" of the Microscopy Innocore Day that took place at University of Coimbra, Coimbra, Portugal on June 21st, 2022.
Outro
2022/05/24 - 2022/05/25 Participation in the InnoCore Challenge, an initiative developed within the framework of the Project INNOCORE-"Core Technologies for Education and Innovation in Life Sciences" and the Erasmus+ programme for strategic partnerships. Over the course of two months, students received online training in innovation, research, and business trends. The goal was to develop innovative solutions for specific Life Science challenges presented by companies. The final event took place in Granada, Spain, on May 24th-25th, 2022.
Oficina (workshop)
2022/01/18 - 2022/01/19 PDBEB symposium: The overview effect, organized by the Doctoral Programme in Experimental Biology and Biomedicine (PDBEB) students, from the Institute of Interdisciplinary Research, University of Coimbra (IIIUC).
Conferência
 BEB Day 2022: The overview effect
Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal
2021/12/01 - 2021/12/03 XVII Meeting of the Portuguese Society for Neuroscience, which will be held in Coimbra, from December 1st - 3rd, 2021. The meeting provided a dynamic and informal oportunity for scientific exchange in the different areas of Neuroscience.
Conferência
 XVII Meeting of the Portuguese Society for Neuroscience - SPN 2021
2019/03/20 - 2019/03/22 Tissue Clearing Workshop. The main subjects addressed included Tissue Clearing Protocols, Lightsheet Microscopy (Lightsheet Z.1), Multiphoton Microscopy (LSM710 NLO), Confocal Microscopy (LSM 710) and Arivis Software.
Oficina (workshop)
 Tissue Clearing Workshop
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Microscopy Imaging Center of Coimbra (MICC), Portugal
2016/11/18 - 2016/11/19 1st International Symposium - Brain Without Borders: modeling, disease and repair, held in Coimbra, Portugal, on 18-19th November 2016. 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.
Simpósio
 1st International Symposium - Brain Without Borders: modeling, disease and repair
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
2014/10/31 - 2014/11/01 Emerging Pathways for Young Scientists: -Non-traditional career paths for young scientists: scientific policy, communication, enterprise/private-sector, and non-governmental organizations. -Professional development with 2 ASM virtual workshops on Scientific Writing and Publishing and the Art of Science Communications.
Oficina (workshop)
 Emerging Pathways for Young Scientists 2.0 - Including ASM virtual workshops on Scientific Writing and Publishing and the Art of Science Communications
The American Society for Microbiology (ASM), Estados Unidos

Membro de associação

Nome da associação Tipo de participação
2023/12/01 - Atual Member of the Ataxia Global Initiative (AGI). AGI is a worldwide public-private partnership that has the goal to facilitate the clinical development of therapies for ataxias by promoting data sharing, establishing global research standards, providing trial-readiness resources, organising educational events, and offering training and information to researchers, clinicians, patients and the public.
2022/01/01 - Atual Member of the European Society of Gene and Cell Therapy (ESGCT). ESGCT promotes basic and clinical research in gene therapy, cell therapy and genetic vaccines by facilitating education, the exchange of information and technology, and by serving as a professional adviser to stakeholder communities and regulatory bodies in Europe. Student Membership
Distinções

Prémio

2024 Best poster communication award with the work entitled “Allele-specific silencing for Spinocerebellar Ataxia Type 3 via AAV9 polycistronic microRNA-based therapy”, (co-authorship)
2020 Best Basic Research award with the work entitled "Permanent Inactivation of the ATXN3 Gene: A Possible Therapeutic Approach for Machado-Joseph Disease", (co-authorship).
2018 Winner of the BEB/Bluepharma Award, 2nd Edition, for the Best Master's Thesis.
PhD Programme in Experimental Biology and Biomedicine (PDBEB), Portugal

Bluepharma, Portugal
2018 Best work in basic science with the work entitled "Tailoring gene editing strategies for the treatment of Machado-Joseph disease", (co-authorship).
Portuguese Society of Movement Disorders (SPDMov 2018), Portugal

Outra distinção

2022 Publication selected as the Editor's Choice (Gene Therapy Journal). The publication with DOI: 10.1038/s41434-022-00372-z was selected as a key paper that highlights some of the best current research published in the Gene Therapy Journal.
2022 Front cover of the Gene Therapy Journal (Volume 29, Issue 12). Selection of an image of the publication with DOI: 10.1038/s41434-022-00372-z. (https://www.nature.com/gt/volumes/29/issues/12).