Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells

Detalhes bibliográficos
Autor(a) principal: Virgolini, Nikolaus
Data de Publicação: 2023
Idioma: eng
Título da fonte: Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)
Texto Completo: http://hdl.handle.net/10362/153335
Resumo: "The insect cell-baculovirus expression vector system (IC-BEVS) has been established as an important platform for the manufacturing of therapeutical products, including vectors for gene therapy such as recombinant adeno-associated virus (rAAV). Nevertheless, further characterisation of the underlying biological mechanisms of the IC-BEVS as well as improved engineering strategies are needed to further leverage this system towards optimal product titers and quality. In this thesis, transcriptome analysis approaches (bulk and single-cell) were applied to understand the impact on Sf9 cells of a dual baculovirus co-infection process to produce rAAV. In addition, an improved CRISPR-Cas9 delivery and gene knockout strategy was established to enhance the efficiency of target gene knockout in IC-BEVS."
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spelling Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cellsIC-BEVSTranscriptomeGene TherapySf9 CellsVirusDomínio/Área Científica::Ciências Naturais"The insect cell-baculovirus expression vector system (IC-BEVS) has been established as an important platform for the manufacturing of therapeutical products, including vectors for gene therapy such as recombinant adeno-associated virus (rAAV). Nevertheless, further characterisation of the underlying biological mechanisms of the IC-BEVS as well as improved engineering strategies are needed to further leverage this system towards optimal product titers and quality. In this thesis, transcriptome analysis approaches (bulk and single-cell) were applied to understand the impact on Sf9 cells of a dual baculovirus co-infection process to produce rAAV. In addition, an improved CRISPR-Cas9 delivery and gene knockout strategy was established to enhance the efficiency of target gene knockout in IC-BEVS."Associate Laboratory LS4FUTURE A/P/0087/2020Alves, PaulaRoldão, AntónioIsidro, InêsRUNVirgolini, Nikolaus2023-04-132023-042026-03-31T00:00:00Z2023-04-13T00:00:00Zdoctoral thesisinfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10362/153335TID:101739834enginfo:eu-repo/semantics/embargoedAccessreponame:Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)instname:FCCN, serviços digitais da FCT – Fundação para a Ciência e a Tecnologiainstacron:RCAAP2024-05-22T18:11:46Zoai:run.unl.pt:10362/153335Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireinfo@rcaap.ptopendoar:https://opendoar.ac.uk/repository/71602025-05-28T17:42:14.023414Repositórios Científicos de Acesso Aberto de Portugal (RCAAP) - FCCN, serviços digitais da FCT – Fundação para a Ciência e a Tecnologiafalse
dc.title.none.fl_str_mv Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
title Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
spellingShingle Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
Virgolini, Nikolaus
IC-BEVS
Transcriptome
Gene Therapy
Sf9 Cells
Virus
Domínio/Área Científica::Ciências Naturais
title_short Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
title_full Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
title_fullStr Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
title_full_unstemmed Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
title_sort Leveraging single cell analysis and genetic engineering to enhance recombinant AAV production in insect cells
author Virgolini, Nikolaus
author_facet Virgolini, Nikolaus
author_role author
dc.contributor.none.fl_str_mv Alves, Paula
Roldão, António
Isidro, Inês
RUN
dc.contributor.author.fl_str_mv Virgolini, Nikolaus
dc.subject.por.fl_str_mv IC-BEVS
Transcriptome
Gene Therapy
Sf9 Cells
Virus
Domínio/Área Científica::Ciências Naturais
topic IC-BEVS
Transcriptome
Gene Therapy
Sf9 Cells
Virus
Domínio/Área Científica::Ciências Naturais
description "The insect cell-baculovirus expression vector system (IC-BEVS) has been established as an important platform for the manufacturing of therapeutical products, including vectors for gene therapy such as recombinant adeno-associated virus (rAAV). Nevertheless, further characterisation of the underlying biological mechanisms of the IC-BEVS as well as improved engineering strategies are needed to further leverage this system towards optimal product titers and quality. In this thesis, transcriptome analysis approaches (bulk and single-cell) were applied to understand the impact on Sf9 cells of a dual baculovirus co-infection process to produce rAAV. In addition, an improved CRISPR-Cas9 delivery and gene knockout strategy was established to enhance the efficiency of target gene knockout in IC-BEVS."
publishDate 2023
dc.date.none.fl_str_mv 2023-04-13
2023-04
2023-04-13T00:00:00Z
2026-03-31T00:00:00Z
dc.type.driver.fl_str_mv doctoral thesis
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10362/153335
TID:101739834
url http://hdl.handle.net/10362/153335
identifier_str_mv TID:101739834
dc.language.iso.fl_str_mv eng
language eng
dc.rights.driver.fl_str_mv info:eu-repo/semantics/embargoedAccess
eu_rights_str_mv embargoedAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)
instname:FCCN, serviços digitais da FCT – Fundação para a Ciência e a Tecnologia
instacron:RCAAP
instname_str FCCN, serviços digitais da FCT – Fundação para a Ciência e a Tecnologia
instacron_str RCAAP
institution RCAAP
reponame_str Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)
collection Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)
repository.name.fl_str_mv Repositórios Científicos de Acesso Aberto de Portugal (RCAAP) - FCCN, serviços digitais da FCT – Fundação para a Ciência e a Tecnologia
repository.mail.fl_str_mv info@rcaap.pt
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