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dc.contributor.author
Pascual-Pasto, Guillem  
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Bazan-Peregrino, Miriam  
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Olaciregui, Nagore G.  
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Restrepo Perdomo, Camilo A.  
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Mato Berciano, Ana  
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Ottaviani, Daniela  
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Weber, Klaus  
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Correa, Genoveva  
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Paco, Sonia  
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Vila Ubach, Monica  
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Cuadrado Vilanova, Maria  
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Castillo Ecija, Helena  
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Botteri, Gaia  
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Garcia Gerique, Laura  
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Moreno Gilabert, Helena  
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Gimenez Alejandre, Marta  
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Alonso Lopez, Patricia  
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Farrera Sal, Marti  
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Torres Manjon, Silvia  
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Ramos Lozano, Dolores  
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Moreno, Rafael  
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Aerts, Isabelle  
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Doz, François  
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Cassoux, Nathalie  
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Chapeaublanc, Elodie  
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Torrebadell, Montserrat  
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Roldan, Monica  
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König, Andrés  
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Suñol, Mariona  
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Claverol, Joana  
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Lavarino, Cinzia  
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De Torres, Carmen  
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Fu, Ligia  
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Radvanyi, François  
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Munier, Francis L.  
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Catalá-Mora, Jaume  
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Mora, Jaume  
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Alemany, Ramón  
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Cascalló, Manel  
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Chantada, Guillermo Luis  
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Montero Carcaboso, Angel  
dc.date.available
2020-11-17T15:21:02Z  
dc.date.issued
2019-01  
dc.identifier.citation
Pascual-Pasto, Guillem; Bazan-Peregrino, Miriam; Olaciregui, Nagore G.; Restrepo Perdomo, Camilo A.; Mato Berciano, Ana; et al.; Therapeutic targeting of the RB1 pathway in retinoblastoma with the oncolytic adenovirus VCN-01; American Association for the Advancement of Science; Science Translational Medicine; 11; 476; 1-2019; 1-12  
dc.identifier.issn
1946-6234  
dc.identifier.uri
http://hdl.handle.net/11336/118519  
dc.description.abstract
Retinoblastoma is a pediatric solid tumor of the retina activated upon homozygous inactivation of the tumor suppressor RB1. VCN-01 is an oncolytic adenovirus designed to replicate selectively in tumor cells with high abundance of free E2F-1, a consequence of a dysfunctional RB1 pathway. Thus, we reasoned that VCN-01 could provide targeted therapeutic activity against even chemoresistant retinoblastoma. In vitro, VCN-01 effectively killed patient-derived retinoblastoma models. In mice, intravitreous administration of VCN-01 in retinoblastoma xenografts induced tumor necrosis, improved ocular survival compared with standard-of-care chemotherapy, and prevented micrometastatic dissemination into the brain. In juvenile immunocompetent rabbits, VCN-01 did not replicate in retinas, induced minor local side effects, and only leaked slightly and for a short time into the blood. Initial phase 1 data in patients showed the feasibility of the administration of intravitreous VCN-01 and resulted in antitumor activity in retinoblastoma vitreous seeds and evidence of viral replication markers in tumor cells. The treatment caused local vitreous inflammation but no systemic complications. Thus, oncolytic adenoviruses targeting RB1 might provide a tumor-selective and chemotherapy-independent treatment option for retinoblastoma.  
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application/pdf  
dc.language.iso
eng  
dc.publisher
American Association for the Advancement of Science  
dc.rights
info:eu-repo/semantics/restrictedAccess  
dc.rights.uri
https://creativecommons.org/licenses/by-nc-sa/2.5/ar/  
dc.subject
RETINOBLASTOMA  
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ONCOLYTIC VIRUS  
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E2F  
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TARGETED THERAPY  
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Bioquímica y Biología Molecular  
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Medicina Básica  
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CIENCIAS MÉDICAS Y DE LA SALUD  
dc.title
Therapeutic targeting of the RB1 pathway in retinoblastoma with the oncolytic adenovirus VCN-01  
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info:eu-repo/semantics/article  
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info:ar-repo/semantics/artículo  
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info:eu-repo/semantics/publishedVersion  
dc.date.updated
2020-10-29T20:04:38Z  
dc.identifier.eissn
1946-6242  
dc.journal.volume
11  
dc.journal.number
476  
dc.journal.pagination
1-12  
dc.journal.pais
Estados Unidos  
dc.description.fil
Fil: Pascual-Pasto, Guillem. Hospital Sant Joan de Déu; España  
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Fil: Bazan-Peregrino, Miriam. No especifíca;  
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Fil: Olaciregui, Nagore G.. Hospital Sant Joan de Déu; España  
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Fil: Restrepo Perdomo, Camilo A.. Hospital Sant Joan de Déu; España  
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Fil: Mato Berciano, Ana. No especifíca;  
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Fil: Ottaviani, Daniela. Centre National de la Recherche Scientifique; Francia  
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Fil: Weber, Klaus. No especifíca;  
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Fil: Correa, Genoveva. Hospital Sant Joan de Déu; España  
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Fil: Paco, Sonia. Hospital Sant Joan de Déu; España  
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Fil: Vila Ubach, Monica. Hospital Sant Joan de Déu; España  
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Fil: Cuadrado Vilanova, Maria. Hospital Sant Joan de Déu; España  
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Fil: Castillo Ecija, Helena. Hospital Sant Joan de Déu; España  
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Fil: Botteri, Gaia. Hospital Sant Joan de Déu; España  
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Fil: Garcia Gerique, Laura. Hospital Sant Joan de Déu; España  
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Fil: Moreno Gilabert, Helena. Hospital Sant Joan de Déu; España  
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Fil: Gimenez Alejandre, Marta. No especifíca;  
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Fil: Alonso Lopez, Patricia. No especifíca;  
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Fil: Farrera Sal, Marti. No especifíca;  
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Fil: Torres Manjon, Silvia. Instituto de Investigación Biomédica de Bellvitge; España  
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Fil: Ramos Lozano, Dolores. Instituto de Investigación Biomédica de Bellvitge; España  
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Fil: Moreno, Rafael. Instituto de Investigación Biomédica de Bellvitge; España  
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Fil: Aerts, Isabelle. Centre National de la Recherche Scientifique; Francia  
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Fil: Doz, François. Universite Paris Descartes; Francia. Centre National de la Recherche Scientifique; Francia  
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Fil: Cassoux, Nathalie. Centre National de la Recherche Scientifique; Francia. Universite Paris Descartes; Francia  
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Fil: Chapeaublanc, Elodie. Centre National de la Recherche Scientifique; Francia  
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Fil: Torrebadell, Montserrat. Hospital Sant Joan de Déu; España  
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Fil: Roldan, Monica. Hospital Sant Joan de Déu; España  
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Fil: König, Andrés. No especifíca;  
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Fil: Suñol, Mariona. Hospital Sant Joan de Déu; España  
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Fil: Claverol, Joana. Hospital Sant Joan de Déu; España  
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Fil: Lavarino, Cinzia. Hospital Sant Joan de Déu; España  
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Fil: De Torres, Carmen. Hospital Sant Joan de Déu; España  
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Fil: Fu, Ligia. Hospital Escuela Universitario; Honduras  
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Fil: Radvanyi, François. Centre National de la Recherche Scientifique; Francia  
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Fil: Munier, Francis L.. Hopital Ophtalmique Jules Gonin; Suiza  
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Fil: Catalá-Mora, Jaume. Hospital Sant Joan de Déu; España  
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Fil: Mora, Jaume. Hospital Sant Joan de Déu; España  
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Fil: Alemany, Ramón. Instituto de Investigación Biomédica de Bellvitge; España  
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Fil: Cascalló, Manel. No especifíca;  
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Fil: Chantada, Guillermo Luis. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentina. Gobierno de la Ciudad de Buenos Aires. Hospital de Pediatría "Juan P. Garrahan"; Argentina  
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Fil: Montero Carcaboso, Angel. Hospital Sant Joan de Déu; España  
dc.journal.title
Science Translational Medicine  
dc.relation.alternativeid
info:eu-repo/semantics/altIdentifier/doi/http://dx.doi.org/10.1126/scitranslmed.aat9321  
dc.relation.alternativeid
info:eu-repo/semantics/altIdentifier/url/https://stm.sciencemag.org/content/11/476/eaat9321