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Please use this identifier to cite or link to this item: https://accedacris.ulpgc.es/jspui/handle/10553/139828
DC FieldValueLanguage
dc.contributor.authorBance, M.en_US
dc.contributor.authorLustig, L.en_US
dc.contributor.authorIshiyama, A.en_US
dc.contributor.authorNash, R.en_US
dc.contributor.authorPolo, R.en_US
dc.contributor.authorRamos De Miguel, Ángelen_US
dc.contributor.authorManrique, M. J.en_US
dc.contributor.authorRubinstein, J. T.en_US
dc.contributor.authorLandry, E.en_US
dc.contributor.authorRunge, C.en_US
dc.contributor.authorQuigley, T.en_US
dc.contributor.authorRiggs, J.en_US
dc.contributor.authorCorrales, E.en_US
dc.contributor.authorWhitton, J.en_US
dc.contributor.authorAnderson, J.en_US
dc.contributor.authorValayannopoulos, V.en_US
dc.date.accessioned2025-06-10T12:23:52Z-
dc.date.available2025-06-10T12:23:52Z-
dc.date.issued2025en_US
dc.identifier.issn1043-0342en_US
dc.identifier.otherWoS-
dc.identifier.urihttps://accedacris.ulpgc.es/handle/10553/139828-
dc.description.abstractOtoferlin is critical for inner hair cell (IHC) signal transmission to auditory nerve fibers. Biallelic otoferlin gene (OTOF) variants typically cause severe-to-profound deafness. As per preclinical data, DB-OTO gene therapy promotes IHC-selective otoferlin expression from the human OTOF gene, which may instate high-quality hearing. In this first-in-human multicenter, phase 1/2 open-label clinical trial with DB-OTO (CHORD, NCT05788536), safety, tolerability and preliminary efficacy of DB-OTO is evaluated in pediatric patients with profound OTOF-related deafness.en_US
dc.languageengen_US
dc.relation.ispartofHuman Gene Therapyen_US
dc.sourceHuman Gene Therapy [ISSN 1043-0342],v. 36 (3-4), p. E136, Abstract OR016, (Febrero 2025)en_US
dc.subject320507 Neurologíaen_US
dc.titlePreliminary safety and efficacy of DB-OTO gene therapy in pediatric patients with profound deafness due to otoferlin variants: The CHORD phase 1/2 open-label trialen_US
dc.typeinfo:eu-repo/semantics/conferenceObjecten_US
dc.typeConferenceObjecten_US
dc.relation.conferenceESGCT 31st Annual Congress In collaboration with SITGEC Rome, Italy October 22-25, 2024 Online Onlyen_US
dc.identifier.isi001418811501016-
dc.identifier.eissn1557-7422-
dc.description.lastpageE136en_US
dc.identifier.issue3-4-
dc.description.firstpageE136en_US
dc.relation.volume36en_US
dc.investigacionCiencias de la Saluden_US
dc.type2Actas de congresosen_US
dc.contributor.daisngid19564780-
dc.contributor.daisngid25602827-
dc.contributor.daisngid9385194-
dc.contributor.daisngid26820450-
dc.contributor.daisngid71252153-
dc.contributor.daisngid74837913-
dc.contributor.daisngid74269492-
dc.contributor.daisngid13857597-
dc.contributor.daisngid68832311-
dc.contributor.daisngid54896241-
dc.contributor.daisngid45308696-
dc.contributor.daisngid53704999-
dc.contributor.daisngid50786092-
dc.contributor.daisngid4900948-
dc.contributor.daisngid74861815-
dc.contributor.daisngid30426419-
dc.description.numberofpages1en_US
dc.utils.revisionSíen_US
dc.contributor.wosstandardWOS:Bance, M-
dc.contributor.wosstandardWOS:Lustig, L-
dc.contributor.wosstandardWOS:Ishiyama, A-
dc.contributor.wosstandardWOS:Nash, R-
dc.contributor.wosstandardWOS:Polo, R-
dc.contributor.wosstandardWOS:Ramos, A-
dc.contributor.wosstandardWOS:Manrique, MJ-
dc.contributor.wosstandardWOS:Rubinstein, JT-
dc.contributor.wosstandardWOS:Landry, E-
dc.contributor.wosstandardWOS:Runge, C-
dc.contributor.wosstandardWOS:Quigley, T-
dc.contributor.wosstandardWOS:Riggs, J-
dc.contributor.wosstandardWOS:Corrales, E-
dc.contributor.wosstandardWOS:Whitton, J-
dc.contributor.wosstandardWOS:Anderson, J-
dc.contributor.wosstandardWOS:Valayannopoulos, V-
dc.date.coverdateFebrero 2025en_US
dc.identifier.abstractidOR016-
dc.identifier.ulpgcSíen_US
dc.contributor.buulpgcBU-MEDen_US
dc.description.sjr1,137
dc.description.jcr4,0
dc.description.sjrqQ2
dc.description.jcrqQ1
dc.description.scieSCIE
dc.description.miaricds11,0
item.grantfulltextopen-
item.fulltextCon texto completo-
crisitem.author.deptGIR SIANI: Modelización y Simulación Computacional-
crisitem.author.deptIU de Sistemas Inteligentes y Aplicaciones Numéricas en Ingeniería-
crisitem.author.orcid0000-0002-0528-815X-
crisitem.author.parentorgIU de Sistemas Inteligentes y Aplicaciones Numéricas en Ingeniería-
crisitem.author.fullNameRamos De,Ángel-
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