Models to study basic and applied aspects of lysosomal storage disorders
Autor(a) principal: | |
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Data de Publicação: | 2022 |
Outros Autores: | , |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
Texto Completo: | http://hdl.handle.net/10451/54849 |
Resumo: | © 2022 The Author(s). Published by Elsevier B.V. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
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Models to study basic and applied aspects of lysosomal storage disordersAnimal modelsBBBLysosomesOmicsOptogeneticsOrganoidsStorage disordersiPSCs© 2022 The Author(s). Published by Elsevier B.V. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).The lack of available treatments and fatal outcome in most lysosomal storage disorders (LSDs) have spurred research on pathological mechanisms and novel therapies in recent years. In this effort, experimental methodology in cellular and animal models have been developed, with aims to address major challenges in many LSDs such as patient-to-patient variability and brain condition. These techniques and models have advanced knowledge not only of LSDs but also for other lysosomal disorders and have provided fundamental insights into the biological roles of lysosomes. They can also serve to assess the efficacy of classical therapies and modern drug delivery systems. Here, we summarize the techniques and models used in LSD research, which include both established and recently developed in vitro methods, with general utility or specifically addressing lysosomal features. We also review animal models of LSDs together with cutting-edge technology that may reduce the need for animals in the study of these devastating diseases.This work was supported by the Spanish Ministry of Science and Innovation (PID2020-112830RB-I00) (AEI/FEDER, UE) and the NextGeneration EU-CSIC funds (NeuroAging)ElsevierRepositório da Universidade de LisboaGaudioso, ÁngelSilva, Teresa P.Dolores Ledesma, María2022-10-21T14:40:07Z20222022-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10451/54849engAdv Drug Deliv Rev. 2022 Nov;190:1145320169-409X10.1016/j.addr.2022.1145321872-8294info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-11-08T17:01:30Zoai:repositorio.ul.pt:10451/54849Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T22:05:37.015700Repositório 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 |
Models to study basic and applied aspects of lysosomal storage disorders |
title |
Models to study basic and applied aspects of lysosomal storage disorders |
spellingShingle |
Models to study basic and applied aspects of lysosomal storage disorders Gaudioso, Ángel Animal models BBB Lysosomes Omics Optogenetics Organoids Storage disorders iPSCs |
title_short |
Models to study basic and applied aspects of lysosomal storage disorders |
title_full |
Models to study basic and applied aspects of lysosomal storage disorders |
title_fullStr |
Models to study basic and applied aspects of lysosomal storage disorders |
title_full_unstemmed |
Models to study basic and applied aspects of lysosomal storage disorders |
title_sort |
Models to study basic and applied aspects of lysosomal storage disorders |
author |
Gaudioso, Ángel |
author_facet |
Gaudioso, Ángel Silva, Teresa P. Dolores Ledesma, María |
author_role |
author |
author2 |
Silva, Teresa P. Dolores Ledesma, María |
author2_role |
author author |
dc.contributor.none.fl_str_mv |
Repositório da Universidade de Lisboa |
dc.contributor.author.fl_str_mv |
Gaudioso, Ángel Silva, Teresa P. Dolores Ledesma, María |
dc.subject.por.fl_str_mv |
Animal models BBB Lysosomes Omics Optogenetics Organoids Storage disorders iPSCs |
topic |
Animal models BBB Lysosomes Omics Optogenetics Organoids Storage disorders iPSCs |
description |
© 2022 The Author(s). Published by Elsevier B.V. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
publishDate |
2022 |
dc.date.none.fl_str_mv |
2022-10-21T14:40:07Z 2022 2022-01-01T00:00:00Z |
dc.type.status.fl_str_mv |
info:eu-repo/semantics/publishedVersion |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
http://hdl.handle.net/10451/54849 |
url |
http://hdl.handle.net/10451/54849 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
Adv Drug Deliv Rev. 2022 Nov;190:114532 0169-409X 10.1016/j.addr.2022.114532 1872-8294 |
dc.rights.driver.fl_str_mv |
info:eu-repo/semantics/openAccess |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
application/pdf |
dc.publisher.none.fl_str_mv |
Elsevier |
publisher.none.fl_str_mv |
Elsevier |
dc.source.none.fl_str_mv |
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Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação |
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RCAAP |
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RCAAP |
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Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
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Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
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Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação |
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