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Ana Carolina Silva has a Bachelor’s degree in Biomedical Sciences from the University of Algarve (2017) and a Master's Degree in Molecular and Cellular Biology from the University of Coimbra (2019). During her master's degree she carried out her thesis research project in the Center for Neuroscience and Cell Biology (CNC) of the University of Coimbra, entitled "Alelle-specific silencing of Machado-Joseph disease", whose oral defense led to a final classification of 20 (out of 20). Of note, she was awarded merit scholarships by the University of Algarve and University of Coimbra for her academic accomplishments during her bachelor’s and master’s degrees. Since then, she has been a researcher at CNC-UC at the Gene and Stem Cell Therapies for the Brain Group, where she has participated in different research projects, including projects in collaboration with international pharmaceutical companies (WAVE Life Sciences, UniQure and PTC Therapeutics), with particular focus on the use of antisense oligonucleotides and viral vectors for the treatment of polyglutamine disorders. Currently, she is a PhD student in the doctoral program for Experimental Biology and Biomedicine of the University of Coimbra with a research project that aims at developing an allele-specific microRNA-based therapy encoded by AAV vectors for the treatment of Spinocerebellar Ataxia Type 3. This allowed development of extensive expertise in gene therapy design, viral vector production, and advanced molecular biology techniques, as well as in the application of therapeutic strategies in both in vitro and in vivo disease models. Until now, Ana Carolina has contributed to work that led to 8 publications in international peer-reviewed journals and 20 scientific communications: 9 oral communications and 13 poster communications in national and international scientific meetings. To date, she has contributed to 5 publications in peer-reviewed journals and 25 scientific communications (10 oral and 15 posters).
Identificação

Identificação pessoal

Nome completo
Ana Carolina Pinheiro da Silva

Nomes de citação

  • Silva, Ana Carolina
  • Silva, Ana C

Identificadores de autor

Ciência ID
D31D-2175-A128
ORCID iD
0000-0003-3888-5847

Endereços de correio eletrónico

  • acsilva@cnc.uc.pt (Profissional)

Telefones

Telemóvel
  • 963127747 (Pessoal)

Moradas

  • Rua Larga, Faculty of Medicine, 3004-504 , Coimbra, Coimbra, Portugal (Profissional)

Websites

  • cnc.uc.pt/people/acsilva (Profissional)

Idiomas

Idioma Conversação Leitura Escrita Compreensão Peer-review
Português (Idioma materno)
Inglês Utilizador proficiente (C1) Utilizador proficiente (C1) Utilizador proficiente (C1) Utilizador proficiente (C1) Utilizador proficiente (C1)
Formação
Grau Classificação
2020 - 2025
Em curso
Doctoral Program in Experimental Biology and Biomedicine (Doutoramento)
Universidade de Coimbra Instituto de Investigação Interdisciplinar, Portugal
2022/05/30 - 2022/06/03
Concluído
Biotech Transfer: Step-by-Step (Outros)
Center for Neuroscience and Cell Biology University of Coimbra, Portugal
2020/11/09 - 2020/11/20
Concluído
IV Modular Training in Laboratory Animal Science Function A (Outros)
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Coimbra Institute for Clinical and Biomedical Research, Portugal
16,9
2020/11/09 - 2020/11/20
Concluído
IV Modular Training in Laboratory Animal Science Modules 20, 21, 22 (Outros)
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Coimbra Institute for Clinical and Biomedical Research, Portugal
15,9
2017/09 - 2019/09
Concluído
Molecular and Cellular Biology (Mestrado)
Universidade de Coimbra Faculdade de Ciencias e Tecnologia, Portugal
"Allele-specific silencing of Machado-Joseph disease" (TESE/DISSERTAÇÃO)
19
2014/09 - 2017/07
Concluído
Biomedical Sciences (Licenciatura)
Universidade do Algarve Departamento de Ciências Biomédicas e Medicina, Portugal
"Estudo da regulação da expressão do gene HoxB9 pelo relógio molecular embrionário" (TESE/DISSERTAÇÃO)
18
2016/08/03 - 2016/08/31
Concluído
Internship (Outros)
Universidade dos Açores Departamento de Oceanografia e Pescas, Portugal
2015/07/13 - 2015/08/31
Concluído
Internship (Outros)
Universidade dos Açores Departamento de Oceanografia e Pescas, Portugal
Percurso profissional

Ciência

Categoria Profissional
Instituição de acolhimento
Empregador
2019/11 - Atual Investigador (Investigação) Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Projetos

Bolsa

Designação Financiadores
2020/12/01 - 2024/11/30 Development of an AAV-mediated allele-specific microRNA-based therapy for Machado-Joseph Disease
2020.07721.BD
Bolseiro de Doutoramento
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal

Universidade de Coimbra Centro de Inovação em Biomedicina e Biotecnologia, Portugal
Fundação para a Ciência e a Tecnologia
Em curso
2020/05 - 2020/11 Research4COVID-19 - Expedited validation and large-scale manufacture of a blowing device for collection with inactivation of samples for detection of SARS-CoV-2
269_596884125
Investigador
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Fundação para a Ciência e a Tecnologia
Em curso
2019/11 - 2020/11 BrainHealth 2020 - Detecção Precoce, Neuro-modulação e Terapias Avançadas para Neuropatologias
CENTRO-01-0145-FEDER-000008
Bolseiro de Investigação
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Centro 2020
Concluído

Outro

Designação Financiadores
2025/07/01 - Atual CiNTech – technological hub for innovation, translation and industrialization of complex injectable drugs
02/C05-i01.01/2022.PC644865576-00000005
Bolseiro de Investigação
Universidade de Coimbra, Portugal
Em curso
2020/12/01 - Atual miRSilencing
CPD0020A02#IV0020
Bolseiro de Doutoramento
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
PTC Therapeutics Inc
Em curso
2019/11 - Atual Targeting mutant ATXN3 for the treatment of Spinocerebellar Ataxia 3 (SCA3)
.
Bolseiro de Investigação
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
Wave Life Sciences
Em curso
2024/12/01 - 2025/06/30 ARDAT - Improving AAV gene therapy vetor efficiency
Grant agreement ID 945473
Bolseiro de Investigação
Universidade de Coimbra Centro de Neurociências e Biologia Celular, Portugal
European Commission
Concluído
2020/03/26 - 2020/05 Laboratório de Análises Clínicas da Universidade de Coimbra - SARS-Cov2 Detection
NA.
Investigador
Universidade de Coimbra Laboratório de Análises Clínicas , Portugal
Concluído
Produções

Publicações

Artigo em jornal
  1. Vinhas, Ana Catarina; Vasconcelos-Ferreira, Ana; Henriques, Carina S.; Silva, Ana Carolina; et. al. "Uma viagem rara", PÚBLICO, 2020, https://www.publico.pt/2020/02/28/ciencia/fotogaleria/viagem-rara-400481.
Artigo em revista
  1. Kevin Leandro; David Rufino-Ramos; Sara M. Lopes; Francisca Santos Silva; Paulo Rodrigues-Santos; Ana Carolina Silva; Ana Rita Fernandes; et al. "Extracellular vesicles-mediated delivery of SpCas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3". Biomaterials (2026): https://doi.org/10.1016/j.biomaterials.2026.124119.
    10.1016/j.biomaterials.2026.124119
  2. 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 34 1 (2026): 161-178. https://doi.org/10.1016/j.ymthe.2025.10.022.
    Publicado • 10.1016/j.ymthe.2025.10.022
  3. 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
  4. de Sousa-Lourenço, João; Silva, Ana Carolina; Pereira de Almeida, L; Nobre, Rui Jorge. "Molecular therapy for polyQ disorders: from bench to clinical trials". Trends in Molecular Medicine 30 9 (2024): 804-808. https://doi.org/10.1016/j.molmed.2024.05.004.
    Publicado • 10.1016/j.molmed.2024.05.004
  5. Lopes, Miguel M.; Lopes, Sara M.; Baganha, Rafael; Henriques, Carina; Silva, Ana C.; Lobo, Diana D.; Cortes, Luísa; de Almeida, Luís Pereira; 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.
    Publicado • 10.3791/66550
  6. Carina Henriques; Miguel M Lopes; Silva, Ana Carolina; Diana D Lobo; Romina Aron Badin; Philippe Hantraye; Luís Pereira de Almeida; Rui Jorge Nobre. "Viral-based animal models in polyglutamine disorders". Brain (2024): https://doi.org/10.1093/brain/awae012.
    Publicado • 10.1093/brain/awae012
  7. Nobre, Rui Jorge; Lobo, Diana D.; Henriques, Carina; Duarte, Sonia P.; Lopes, Sara M.; Silva, Ana C.; Lopes, Miguel M.; et al. "miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3". Nucleic Acid Therapeutics 32 3 (2022): 194-205. https://doi.org/10.1089/nat.2021.0020.
    Publicado • 10.1089/nat.2021.0020
  8. Silva, Ana C; Lobo, Diana D; Martins, Inês M; Lopes, Sara M; 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 (2019): 407-429. https://doi.org/10.1093/brain/awz328.
    10.1093/brain/awz328
Poster em conferência
  1. Pereira, Ana Maria; Pires, Mariana; Tomás, Inês; Silva, Ana Carolina; Almeida, Rita; Fernandes, Ana Rita; Pena, Frederico; et al. "Engineering RNA-Targeting CRISPR/Cas13 For Allele-Specific ATXN3 Knockdown For Treatment Of Spinocerebellar Ataxia Type 3". Trabalho apresentado em 29th Annual Meeting of the Portuguese Society of Human Genetics, 2025.
  2. Gabriel, Guilherme L; Silva, Marta; Batista, Alexandre; Leandro, Kevin; Almeida, Rita; Silva, Ana Carolina; Miguel M. Lopes; et al. "Hacking Biology: Optimizing AAV-Based Gene Delivery for Enhanced Region-Specific Brain Targeting". Trabalho apresentado em European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, 2024.
  3. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Miguel M. Lopes; 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 European Society for Gene and Cell Therapy (ESGCT) 31st Annual Congress, 2024.
  4. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Lopes, Miguel M; Pereira, Dina; Duarte, Sónia P; 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.
  5. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Miguel M. Lopes; Leandro, Kevin; Pereira, Dina; 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 and Cell Therapy (ASGCT ) Annual Meeting, 2024.
  6. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Fernandes, Ana Rita; Miguel M. Lopes; Pereira, Dina; Duarte, Sónia 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". Trabalho apresentado em European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress, 2023.
  7. Lobo, Diana D; Quintino, Luis; Silva, Ana Carolina; Henriques, Carina; Ribeiro, R; Lopes, Sara M; Perdigão, Pedro; et al. "CRISPR activation as a therapeutic tool for Spinocerebellar ataxia type 3 (SCA3)". Trabalho apresentado em European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress, 2023.
  8. Leandro, Kevin; Rufino-Ramos, David; Sara M. Lopes; Pasqua, Carlo; Santos, Paulo; Silva, Ana Carolina; Lobo, Diana Duarte; et al. "Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease/Spinocerebellar Ataxia Type 3". Trabalho apresentado em European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress, 2023.
  9. Silva, Ana Carolina; Henriques, Carina; Lobo, Diana Duarte; Pereira, Dina; Duarte, Sonia; 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)". Trabalho apresentado em European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress, 2023.
  10. Leandro, Kevin; Rufino-Ramos, David; Sara M. Lopes; Pasqua, Carlo; Santos, Paulo; Silva, Ana Carolina; Lobo, Diana Duarte; et al. "Extracellular vesicles-mediated delivery of CRISPR/Cas9 systems to treat Machado-Joseph Disease". Trabalho apresentado em CAG Triplet Repeat Disorder - Gordon Research Seminar, 2023.
  11. Henriques, Carina; Lopes, Miguel; Albuquerque, Patricia; Rufini-Ramos, David; Gaspar, Laetitia; Lobo, Diana; Silva, Ana Carolina; et al. "Production and isolation of natural and biologically active exo-AAVs for brain gene delivery". Trabalho apresentado em Anual congress of the European Society of Gene and Cell Therapy, 2022.
  12. Silva, Ana Carolina; Lobo, Diana Duarte; Henriques, Carina; Martins, Inês M; Lopes, Sara M. ; Duarte, Sónia; Wagner, Elizabeth; Nobre, Rui Jorge; Pereira de Almeida, Luis. "Silencing of mutant ATXN3 through antisense oligonucleotides rescues molecular and neuropathological features in Machado-Joseph disease mice". Trabalho apresentado em European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress, 2022.
  13. 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. "Antisense oligonucleotide mediated allele-specific silencing of mutant ATXN3 improves disease features in mouse models of Machado-Joseph disease". Trabalho apresentado em XVII Meeting of the Portuguese Society for Neuroscience, 2021.
  14. 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". Trabalho apresentado em 1st Symposium on Oligonucleotide Technologies and Therapeutics in Portugal (OTP2021), 2021.
  15. Afonso, Inês T.; Silva, Ana Carolina; Henriques, Carina; Lopes, Miguel; Baganha, Rafael; Lima, Patricía; Kopenol, Rebekah; Nobre, Rui Jorge. "Brain distribution, spreading and tropism of a recently developed gene delivery system - mixCapAAV1/2vector". Trabalho apresentado em AIMS, 2021.
Resumo em conferência
  1. Afonso, Inês T.; Silva, Ana Carolina; Henriques, Carina; Miguel M. Lopes; Baganha, Rafael; Lima, Patricia; Koppenol, Rebekah; et al. "mixCapAAV1/2 Vector: Distribution, Spreading and Tropism Analysis in Normal Mice". Trabalho apresentado em 24th Annual Meeting of the PortugueseSociety for Human Genetics (SPGH), Lisboa, 2020.
Tese / Dissertação
  1. Silva, Ana Carolina. "Allele-specific silencing of Machado-Joseph disease". Mestrado, Universidade de Coimbra Faculdade de Ciencias e Tecnologia, 2019. https://estudogeral.sib.uc.pt/handle/10316/87992.
  2. Silva, Ana Carolina. "Estudo da regulação da expressão do gene HoxB9 pelo relógio molecular embrionário". Licenciatura, Universidade do Algarve Departamento de Ciências Biomédicas e Medicina, 2017.
Atividades

Apresentação oral de trabalho

Título da apresentação Nome do evento
Anfitrião (Local do evento)
2026/02 Engineering RNA-Targeting CRISPR/Cas13 For Allele-Specific ATXN3 Knockdown For Treatment Of Spinocerebellar Ataxia Type 3 56th Annual Meeting of the Portuguese Society of Pharmacology
Portuguese Society of Pharmacology (Coimrba, Portugal)
2025/09/05 rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3 Innovative Therapies 5th CiBB Retreat
CiBB - Centre for Innovative Biomedicine and Biotechnology (Coimbra, Portugal)
2024/11 Allele-specific silencing of mutant ataxin-3 via single administration of AAV9 vectors mitigates neuropathology and motor deficits in Spinocerebellar Ataxia Type 3 International Congress for Ataxia Research (ICAR) 2024
International Congress for Ataxia Research (ICAR) (London, Reino Unido)
2024/02/09 Targeted silencing via AAV9 polycistronic microRNA therapy rescues SCA3 molecular, neuropathological and motor deficits 54th Annual meeting of the Portuguese Pharmacology Society
Portuguese Pharmacology Society (Lisboa, Portugal)
2024/02 Extracellular vesicles delivery of CRISPR-Cas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3 54th Meeting of the Portuguese Society of Pharmacology
Portuguese Society of Pharmacology (Lisbon, Portugal)
2024/02 Hacking biology: optimizing AAV-based gene delivery for enhanced brain region-specific targeting 54th Annual meeting of the Portuguese Pharmacology Society
Portuguese Pharmacology Society (Lisbon, Portugal)
2023/11/24 Polycistronic artificial microRNAs delivered by AAV9 alleviate Spinocerebellar Ataxia Type 3 (SCA3) Pharma PhD Day 2023
(Coimbra, Portugal)
2023/05 Isolation of Natural and Biologically Active EV-AAVs for Brain Gene Delivery American Society of Gene & Cell Therapy (ASGCT) 26th Annual Congress
American Society of Gene & Cell Therapy (ASGCT) (Los Angeles, Estados Unidos)
2021/02/18 MixCapAAV1/2 vector: a novel gene delivery system distribution, spreading and tropism analysis in normal mice LI Annual meeting of the Portuguese Pharmacology Society, XXXIX Clinical Pharmacology meeting and XX Toxicology meeting
2021/02 Allele-specific silencing of Machado-Joseph disease using antisense oligonucleotide therapeutics LI Annual meeting of the Portuguese Pharmacology Society, XXXIX Clinical Pharmacology meeting and XX Toxicology meeting
Portuguese Pharmacology Society (Lisboa, Portugal)

Participação em evento

Descrição da atividade
Tipo de evento
Nome do evento
Instituição / Organização
2023/09/29 - 2023/09/29 Science communication activity during the "Noite Europeia dos Investigadores" in Coimbra (2023 edition).
Outro
Noite Europeia dos Investigadores Coimbra
Noite Europeia dos Investigadores UC, Portugal
2023/05/23 - 2023/05/23 Science communication activity to promote scientific literacy in the Pint of Science Festival (2023 edition).
Festival
Pint of Science
Pint of Science Portugal, Portugal

Membro de associação

Nome da associação Tipo de participação
2022 - Atual European Society of Gene and Cell Therapy Member
2021 - Atual Portuguese Society for Neuroscience Member
Distinções

Prémio

2021 Scholarship to participate in the 2021 meeting of the Portuguese Society for Neuroscience
Sociedade Portuguesa de Neurociências, Portugal
2020 Bolsa por Mérito pela Universidade de Coimbra 2018/2019
2018 Prémio da Caixa Geral de Depósitos de melhor aluna graduada na Universidade do Algarve em Ciências Biomédicas no ano letivo 2016/2017
Caixa Geral de Depositos, Portugal