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dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorKagiava, Alexia
dc.contributor.authorKaraiskos, Christos
dc.contributor.authorLapathitis, George
dc.contributor.authorHeslegrave, Amanda
dc.contributor.authorSargiannidou, Irene
dc.contributor.authorZetterberg, Henrik
dc.contributor.authorBosch, Assumpció
dc.date.accessioned2023-09-01T12:46:31Z
dc.date.available2023-09-01T12:46:31Z
dc.date.issued2023-09-14
dc.identifier.citationKagiava A, Karaiskos C, Lapathitis G, Heslegrave A, Sargiannidou I, Zetterberg H, et al. Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy. Mol Ther Methods Clin Dev. 2023 Sep 14;30:377–93.
dc.identifier.issn2329-0501
dc.identifier.urihttps://hdl.handle.net/11351/10214
dc.descriptionMutacions retingudes per Golgi; Desmielinització; Teràpia de genes
dc.description.sponsorshipThis work was funded by the Muscular Dystrophy Association (MDA) and Charcot-Marie-Tooth Association (CMTA) (grant MDA 603003 to K.A.K.), as well as the UK Dementia Research Institute at UCL (UKDRI-1003 to H.Z. and A.H.).
dc.language.isoeng
dc.publisherCell Press
dc.relation.ispartofseriesMolecular Therapy - Methods & Clinical Development;30
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.sourceScientia
dc.subjectTeràpia genètica
dc.subjectAnomalies cromosòmiques
dc.subjectNeuropatia - Tractament
dc.subject.meshMutation
dc.subject.meshCharcot-Marie-Tooth Disease
dc.subject.meshGenetic Therapy
dc.titleGene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.1016/j.omtm.2023.07.011
dc.subject.decsmutación
dc.subject.decsenfermedad de Charcot-Marie-Tooth
dc.subject.decsterapia genética
dc.relation.publishversionhttps://doi.org/10.1016/j.omtm.2023.07.011
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.organismesInstitut Català de la Salut
dc.contributor.authoraffiliation[Kagiava A, Karaiskos C, Lapathitis G, Sargiannidou I] Neuroscience Department, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular Medicine, Nicosia, Cyprus. [Heslegrave A] Department of Neurodegenerative Disease, UCL Institute of Neurology, London, UK. UK Dementia Research Institute at UCL, London, UK. [Zetterberg H] Department of Neurodegenerative Disease, UCL Institute of Neurology, London, UK. UK Dementia Research Institute at UCL, London, UK. Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, the Sahlgrenska Academy at the University of Gothenburg, Mölndal, Sweden. Clinical Neurochemistry Laboratory, Sahlgrenska University Hospital, Mölndal, Sweden. Hong Kong Center for Neurodegenerative Diseases, Clear Water Bay, Hong Kong, China. Wisconsin Alzheimer’s Disease Research Center, University of Wisconsin School of Medicine and Public Health, University of Wisconsin-Madison, Madison, USA. [Bosch A] Departament de Bioquímica i Biologia Molecular, Institut de Neurociències, Universitat Autònoma de Barcelona, Bellaterra, Spain. Unitat Mixta UAB-VHIR, Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain. Centro de Investigación Biomédica en Red sobre Enfermedades Neurodegenerativas (CIBERNED), Instituto de Salud Carlos III, Madrid, Spain
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


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