Models to study basic and applied aspects of lysosomal storage disorders

Bibliographic Details
Main Author: Gaudioso, Ángel
Publication Date: 2022
Other Authors: Silva, Teresa P., Dolores Ledesma, María
Format: Article
Language: eng
Source: Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)
Download full: http://hdl.handle.net/10451/54849
Summary: © 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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spelling 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:Repositórios Científicos de Acesso Aberto de Portugal (RCAAP)instname:FCCN, serviços digitais da FCT – Fundação para a Ciência e a Tecnologiainstacron:RCAAP2025-03-17T14:50:32Zoai:repositorio.ulisboa.pt:10451/54849Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireinfo@rcaap.ptopendoar:https://opendoar.ac.uk/repository/71602025-05-29T03:26:30.633218Repositórios 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 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 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
_version_ 1833601699136143360

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