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Authordc.contributor.authorValenzuela, Vicente 
Authordc.contributor.authorJackson, Kasey L. 
Authordc.contributor.authorSardi, Sergio P. 
Authordc.contributor.authorHetz Flores, Claudio
Admission datedc.date.accessioned2019-03-18T12:02:11Z
Available datedc.date.available2019-03-18T12:02:11Z
Publication datedc.date.issued2018
Cita de ítemdc.identifier.citationMolecular Therapy, Volumen 26, Issue 6, 2018, Pages 1404-1413
Identifierdc.identifier.issn15250024
Identifierdc.identifier.issn15250016
Identifierdc.identifier.other10.1016/j.ymthe.2018.04.004
Identifierdc.identifier.urihttps://repositorio.uchile.cl/handle/2250/167443
Abstractdc.description.abstract© 2018 The American Society of Gene and Cell Therapy Proteostasis alterations are proposed as a transversal hallmark of several pathological conditions, including metabolic disorders, mechanical injury, cardiac malfunction, neurodegeneration, and cancer. Strategies to improve proteostasis aim to reduce the accumulation of specific disease-related misfolded proteins or bolster the endogenous mechanisms to fold and degrade abnormal proteins. Endoplasmic reticulum (ER) stress is a common pathological signature of a variety of diseases, which engages the unfolded protein response (UPR) as a cellular reaction to mitigate ER stress. Pharmacological modulation of the UPR is challenging considering the physiological importance of the pathway in various organs. However, local targeting of ER stress responses in the affected tissue using gene therapy is emerging as a possible solution to overcome side effects. The delivery of ER chaperones or active UPR components using adeno-associated virus (A
Lenguagedc.language.isoen
Publisherdc.publisherCell Press
Type of licensedc.rightsAttribution-NonCommercial-NoDerivs 3.0 Chile
Link to Licensedc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/3.0/cl/
Sourcedc.sourceMolecular Therapy
Keywordsdc.subjectAAV
Keywordsdc.subjectdiabetes
Keywordsdc.subjectER stress
Keywordsdc.subjectgene therapy
Keywordsdc.subjectneurodegeneration
Keywordsdc.subjectprotein misfolding
Keywordsdc.subjectUPR
Títulodc.titleGene Therapy Strategies to Restore ER Proteostasis in Disease
Document typedc.typeArtículo de revista
dcterms.accessRightsdcterms.accessRightsAcceso Abierto
Catalogueruchile.catalogadorSCOPUS
Indexationuchile.indexArtículo de publicación SCOPUS
uchile.cosechauchile.cosechaSI


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Attribution-NonCommercial-NoDerivs 3.0 Chile
Except where otherwise noted, this item's license is described as Attribution-NonCommercial-NoDerivs 3.0 Chile