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Miguel Monteiro Lopes graduated as a BSc in Biochemistry at the Faculty of Science and Technology from the University of Coimbra in 2015. He then completed his master¿s degree in Biomedical Research at the Faculty of Medicine of the same University in 2017, with excellent mark (19 out of 20). His thesis, entitled AAV-Based Tools for the Development of Cellular and Animal Models of Machado-Joseph Disease, was developed at the Vectors and Gene Therapy group at the Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC) and aimed to generate new models of Machado-Joseph disease based on the use of mosaic recombinant adeno-associated viral vectors. This thesis received the classification of 20/20 for the high-quality work, that was latter distinguished with the BEB/Bluepharma award (2nd edition, 2018) attributed to the best Master thesis developed at CNC-UC. During this period, he successfully completed the theoretical and practical course on Laboratory Animal Sciences designed according to the FELASA recommendations for Category B, receiving the respective authorization by the Portuguese animal health authority (DGAV) to perform procedures on animals (small rodents and fishes). Between November 2017 and December 2019, he participated as a research fellow (POCI-COMPETE POCI-01-0145-FEDER-016807) in the project ImaGene (Imaging Gene Therapy in the Brain, PTDC/BBB-NAN/0932/2014), at the Vectors and Gene Therapy group, CNC-UC. In the framework of this project, he was an invited researcher at the Carl Zeiss AG facility, in Oberkochen, Germany (2019). The generated results were presented in scientific congresses as i) a flash talk (EMBO|EMBL 2019, Germany), ii) posters (EMBO|EMBL 2019, Germany, and 1st Annual Meeting in Pharmaceutical Sciences, 2019, Portugal) and iii) oral communications (SPAOM 2019, Portugal and EMC 2020, Virtual Meeting), including one as an invited speaker in a workshop (SPAOM 2019, Portugal). He also participated as a research fellow in the project BrainHealth 2020 (Detecção precoce, Neuro-modulação e Terapias Avançadas para Neuropatologias, CENTRO-01-0145-FEDER-000008), and has been actively working in the implementation of protocols for the purification of AAVs, in tight collaboration with ViraVector@UC, a core facility for viral production hosted by CNC-UC. Moreover, he also contributed to the development of different projects, that were disseminated in national and international scientific congresses, being the author of a total of 13 posters and 20 oral communications. He is also a co-author of 3 original papers and of 2 works distinguished with the prize of best work in basic science: Tailoring gene editing strategies for the treatment of Machado-Joseph disease (SPDMov2018) and Permanent Inactivation of the ATXN3 Gene: A Possible Therapeutic Approach for Machado-Joseph Disease (SPGH, 2020). In 2020 he ssuccessfully completed two postgraduate courses (not awarding degree) that allowed him to acquire essential complementary tools for scientific research: the training course in Statistics and Multivariate Analysis in R: applications in multicomponent systems (Faculty of Sciences and Technology of the University of Coimbra) and the course of English for Academic Purposes II (Faculty of Arts and Humanities of the University of Coimbra). In December 2020, he enrolled in the international PhD programme in Experimental Biology and Biomedicine, coordinated by CNC/IIIUC. He is currently conducting research on his thesis entitled Enhanced AAV vector toolkit for the generation of novel animal models and therapeutic strategies for polyglutamine diseases, supported by a PhD fellowship from the Foundation for Science and Technology.
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Identification

Personal identification

Full name
Miguel Monteiro Lopes
Gender
Male

Citation names

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

Author identifiers

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

Telephones

Telephone
  • 239820190 (Professional)

Knowledge fields

  • Medical and Health Sciences - Medical Biotechnology - Gene-based Diagnostics and Therapeutic Interventions
  • 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 - Basic Medicine - Neurosciences
  • Medical and Health Sciences - Basic Medicine - Human Genetics
  • Natural sciences - Biological Sciences - Biochemistry
  • Natural sciences - Biological Sciences - Molecular Biology
  • Natural sciences - Biological Sciences - Cell Biology
  • Natural sciences - Biological Sciences - Microbiology

Languages

Language Speaking Reading Writing Listening Peer-review
Portuguese (Mother tongue)
English Advanced (C1) Proficiency (C2) Advanced (C1) Proficiency (C2)
Spanish; Castilian Beginner (A1) Elementary (A2) Beginner (A1) Advanced (C1)
Education
Degree Classification
2020/12/09 - 2025
Ongoing
 Doutoramento em Biologia Experimental e Biomedicina (PDBEB) (Doutoramento)
Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal
2020/03/19 - 2020/06/29
Concluded
 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
Concluded
 Curso de Formação de Inglês para Fins Académicos II (nível intermédio/avançado) (Outros)
Universidade de Coimbra Faculdade de Letras, Portugal
2020/01/02
Concluded
 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
Concluded
 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
Concluded
 Investigação Biomédica (Mestrado)
Major in Neurobiologia
Universidade de Coimbra Faculdade de Medicina, Portugal
"AAV-Based Tools for the Development of Cellular and Animal Models of Machado-Joseph Disease" (THESIS/DISSERTATION)
 19
2017/02/23
Concluded
 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
Concluded
 Bioquímica (Licenciatura)
Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
 14.35
Affiliation

Science

Category
Host institution 		Employer
2020/05/04 - 2021/12/31 Researcher (Research) 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 Researcher (Research) 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 Research Trainee (Research) 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 Research Trainee (Research) 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 Research Trainee (Research) 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
Projects

Grant

Designation Funders
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
PhD Student Fellow
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
Ongoing

Contract

Designation Funders
2022/01/01 - 2023/06/30 Genome editing to rescue Machado-Joseph disease by alternative splicing
EXPL/MED-NEU/0936/2021
Researcher
Universidade de Coimbra, Portugal

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

Fundo Europeu de Desenvolvimento Regional (FEDER)
Concluded
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
Research Fellow
Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, Portugal
 Fundação para a Ciência e a Tecnologia
Concluded

Other

Designation Funders
2020/01/01 - Current Centro de Inovação em Biomedicina e Biotecnologia
UIDB/04539/2020
UIDP/04539/2020
Researcher
Centre for Innovative Biomedicine and Biotechnology (CIBB), Portugal
 Fundação para a Ciência e a Tecnologia
Ongoing
2017/03/01 - Current ViraVector - Viral Vector for Gene Transfer Core Facility
CENTRO-01-0145-FEDER-022095
Researcher
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Ongoing
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
Researcher
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
Concluded
2020/05/04 - 2020/06/04 Covid-19 Lab at CNC / ViraVector
N.A.
Researcher
Concluded
2015/03/01 - 2019/12/31 CNC. IBILI
UID/NEU/04539/2013
UID/NEU/04539/2019
Researcher
CNC.IBILI, Portugal
Concluded
Outputs

Publications

Conference abstract
  1. No authors listed. "Proceedings of the 24th Annual Meeting of the Portuguese Society of Human Genetics (SPGH: Sociedade Portuguesa de Genética Humana)". Paper presented in Portuguese Society of Human Genetics (SPGH), Lisbon, 2021.
    Published • 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. 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. 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". Paper presented in 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  2. 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)". Paper presented in 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  3. 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?". Paper presented in 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  4. 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". Paper presented in 30th Annual Congress of the European Society for Gene and Cell Therapy (ESGCT), 2023.
  5. 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". Paper presented in American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, 2023.
  6. 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.
  7. 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". Paper presented in ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, 2022.
  8. 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". Paper presented in ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, 2022.
  9. 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". Paper presented in ESGCT (European Society for Gene and Cell Therapy) 29th Congress in collaboration with BSGCT, 2022.
  10. 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". Paper presented in 27th European Society for Animal Cell Technology Meeting (ESACT2022), 2022.
  11. 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". Paper presented in 27th European Society for Animal Cell Technology Meeting (ESACT2022), 2022.
  12. 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". Paper presented in XVII Meeting of the Portuguese Society for Neuroscience, 2021.
  13. 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". Paper presented in XVII Meeting of the Portuguese Society for Neuroscience - SPN 2021, 2021.
  14. 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". Paper presented in AIMS Meeting: Annual International (bio)Medical Students Meeting, 2021.
  15. 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". Paper presented in 3rd BEB Day, Winner of the BEB/BluePharma Award, 3rd Edition, 2019.
  16. 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". Paper presented in 1st Annual Meeting in Pharmaceutical Sciences, 2019.
  17. 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". Paper presented in 2019 International Ataxia Research Conference (IARC 2019), 2019.
  18. 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". Paper presented in 2019 International Ataxia Research Conference (IARC 2019), 2019.
  19. 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". Paper presented in 2019 International Machado-Joseph Research Disease Conference, 2019.
  20. 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". Paper presented in 2019 International Machado-Joseph Research Disease Conference, 2019.
  21. 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". Paper presented in 5th GEIVEX Symposium, 2019.
  22. 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". Paper presented in EMBO | EMBL Symposium: Seeing Is Believing – Imaging the Molecular Processes of Life, 2019.
  23. 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". Paper presented in XVI Meeting of the Portuguese Society for Neuroscience (SPN), 2019.
  24. 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". Paper presented in 2nd International Ataxia Research Conference (IARC), 2017.
Journal article
  1. 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.
    10.1093/brain/awae012
  2. 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
  3. 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
  4. 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.
    In press • 10.1016/j.ymthe.2021.07.012
  5. 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
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.
Preprint
  1. Luis Pereira de Almeida; Carina Henriques; Miguel Lopes; Patrícia Albuquerque; David Rufino Ramos; Laetitia Gaspar; Diana Lobo; et al. "Isolation of Biologically Active Extracellular Vesicles-Associated AAVs for Gene Delivery to the Brain by Size Exclusion Chromatography". 2023. https://doi.org/10.21203/rs.3.rs-3220758/v1.
    10.21203/rs.3.rs-3220758/v1
Thesis / Dissertation
  1. Miguel M. Lopes. "AAV-Based Tools For The Development Of Cellular and Animal Models of Machado-Joseph Disease". Master, Grupo de Vectores e Terapia Génica, Centro de Neurociências e Biologia Celular (CNC), Universidade de Coimbra, 2017.

Artistic / Performance

Theatre play
  1. Mário Montenegro; Sara Amaral; João Cardoso; Marta Quatorze; Miguel Lopes; Diana Lobo; Carina Henriques; et al. 2019. Holy CIBB.
Activities

Oral presentation

Presentation title Event name
Host (Event location)
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)
2023/05/31 A novel pipeline for whole-brain biodistribution analysis of AAVs using tissue clearing and light-sheet fluorescence microscopy 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, United States)
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., United States)
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., 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)
2019/11/12 CRISPR-Cas9 Systems Targeting ATXN3 as Therapeutic Approaches for Machado-Joseph Disease 2019 International Machado-Joseph Research Disease Conference
(Washington DC., United States)
2019/11/07 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, Spain)
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)
2019/05/30 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, Italy)

Event participation

Activity description
Type of event 		Event name
Institution / Organization
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.
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.
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.
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.
Other
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.
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).
Conference
 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.
Conference
 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.
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.
Symposium
 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.
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), United States
Distinctions

Award

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

Other distinction

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).