Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines

Detalhes bibliográficos
Autor(a) principal: Lourenço Antunes, Mariana
Data de Publicação: 2023
Tipo de documento: Dissertação
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10362/163343
Resumo: "Recombinant adeno-associated virus (rAAV) vectors are the most promising vectors for gene therapy, as shown by the steady increase of rAAV based gene therapies in the market. Nonetheless, manufacturing clinical-grade rAAVs relies mainly on transient transfection, which is not scalable and necessitates costly raw materials such as GMP-grade DNA. To address this challenge, producer cell lines (PCLs) have emerged as promising alternatives for rAAV production. However, their development is still in the early stages and still requires significant optimization to achieve high yields suitable to the commercial stage demands. By improving the host cell or selection methodology, the cell line generation process can be enhanced.(...)"
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spelling Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell linesrAAVHeLa S3-based systemCRISPR-Cas9 gain-of-function screenGLUL-MSX"Recombinant adeno-associated virus (rAAV) vectors are the most promising vectors for gene therapy, as shown by the steady increase of rAAV based gene therapies in the market. Nonetheless, manufacturing clinical-grade rAAVs relies mainly on transient transfection, which is not scalable and necessitates costly raw materials such as GMP-grade DNA. To address this challenge, producer cell lines (PCLs) have emerged as promising alternatives for rAAV production. However, their development is still in the early stages and still requires significant optimization to achieve high yields suitable to the commercial stage demands. By improving the host cell or selection methodology, the cell line generation process can be enhanced.(...)"Instituto de Tecnologia Química e Biológica António Xavier. Universidade NOVA de lisboaRUNLourenço Antunes, Mariana2023-09-012026-09-30T00:00:00Z2023-09-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/masterThesisapplication/pdfhttp://hdl.handle.net/10362/163343enginfo:eu-repo/semantics/embargoedAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2024-03-11T05:46:32Zoai:run.unl.pt:10362/163343Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T03:59:22.698445Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
title Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
spellingShingle Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
Lourenço Antunes, Mariana
rAAV
HeLa S3-based system
CRISPR-Cas9 gain-of-function screen
GLUL-MSX
title_short Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
title_full Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
title_fullStr Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
title_full_unstemmed Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
title_sort Exploring genetic strategies to boost recombinant adeno associated virus production in HeLa S3 stable producer cell lines
author Lourenço Antunes, Mariana
author_facet Lourenço Antunes, Mariana
author_role author
dc.contributor.none.fl_str_mv RUN
dc.contributor.author.fl_str_mv Lourenço Antunes, Mariana
dc.subject.por.fl_str_mv rAAV
HeLa S3-based system
CRISPR-Cas9 gain-of-function screen
GLUL-MSX
topic rAAV
HeLa S3-based system
CRISPR-Cas9 gain-of-function screen
GLUL-MSX
description "Recombinant adeno-associated virus (rAAV) vectors are the most promising vectors for gene therapy, as shown by the steady increase of rAAV based gene therapies in the market. Nonetheless, manufacturing clinical-grade rAAVs relies mainly on transient transfection, which is not scalable and necessitates costly raw materials such as GMP-grade DNA. To address this challenge, producer cell lines (PCLs) have emerged as promising alternatives for rAAV production. However, their development is still in the early stages and still requires significant optimization to achieve high yields suitable to the commercial stage demands. By improving the host cell or selection methodology, the cell line generation process can be enhanced.(...)"
publishDate 2023
dc.date.none.fl_str_mv 2023-09-01
2023-09-01T00:00:00Z
2026-09-30T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/masterThesis
format masterThesis
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10362/163343
url http://hdl.handle.net/10362/163343
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.publisher.none.fl_str_mv Instituto de Tecnologia Química e Biológica António Xavier. Universidade NOVA de lisboa
publisher.none.fl_str_mv Instituto de Tecnologia Química e Biológica António Xavier. Universidade NOVA de lisboa
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron:RCAAP
instname_str Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron_str RCAAP
institution RCAAP
reponame_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
repository.mail.fl_str_mv
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