Show simple item record

 
dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorMòdol-Caballero, Guillem
dc.contributor.authorHerrando-Grabulosa, Mireia
dc.contributor.authorVerdés Franquesa, Sergi
dc.contributor.authorGarcía-Lareu, Belén
dc.contributor.authorHernández, Neus
dc.contributor.authorFrancos-Quijorna, Isaac
dc.contributor.authorBosch Merino, Assumpció
dc.date.accessioned2022-05-03T12:49:05Z
dc.date.available2022-05-03T12:49:05Z
dc.date.issued2021-09-22
dc.identifier.citationMòdol-Caballero G, Herrando-Grabulosa M, Verdés S, García-Lareu B, Hernández N, Francos-Quijorna I, et al. Gene Therapy Overexpressing Neuregulin 1 Type I in Combination With Neuregulin 1 Type III Promotes Functional Improvement in the SOD1G93A ALS Mice. Front Neurol. 2021 Sep 22;12:693309.
dc.identifier.issn1664-2295
dc.identifier.urihttp://hdl.handle.net/11351/7464
dc.descriptionMotoneuron; Neuregulin 1; Spinal cord
dc.description.abstractAmyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting the neuromuscular system for which currently there is no effective therapy. Motoneuron (MN) degeneration involves several complex mechanisms, including surrounding glial cells and skeletal muscle contributions. Neuregulin 1 (NRG1) is a trophic factor present particularly in MNs and neuromuscular junctions. Our previous studies revealed that gene therapy overexpressing the isoform I (NRG1-I) in skeletal muscles as well as overexpressing the isoform III (NRG1-III) directly in the central nervous system are both effective in preserving MNs in the spinal cord of ALS mice, opening novel therapeutic approaches. In this study, we combined administration of both viral vectors overexpressing NRG1-I in skeletal muscles and NRG1-III in spinal cord of the SOD1G93A mice in order to obtain a synergistic effect. The results showed that the combinatorial gene therapy increased preservation of MNs and of innervated neuromuscular junctions and reduced glial reactivity in the spinal cord of the treated SOD1G93A mice. Moreover, NRG1 isoforms overexpression improved motor function of hindlimb muscles and delayed the onset of clinical disease. However, this combinatory gene therapy did not produce a synergic effect compared with single therapies, suggesting an overlap between NRG1-I and NRG1-III activated pathways and their beneficial effects.
dc.language.isoeng
dc.publisherFrontiers Media
dc.relation.ispartofseriesFrontiers in Neurology;12
dc.rightsAttribution 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/
dc.sourceScientia
dc.subjectEsclerosi lateral amiotròfica - Tractament
dc.subjectTeràpia genètica
dc.subjectRatolins transgènics
dc.subject.meshAmyotrophic Lateral Sclerosis
dc.subject.mesh/therapy
dc.subject.meshGenetic Therapy
dc.subject.meshMice, Transgenic
dc.titleGene Therapy Overexpressing Neuregulin 1 Type I in Combination With Neuregulin 1 Type III Promotes Functional Improvement in the SOD1G93A ALS Mice
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.3389/fneur.2021.693309
dc.subject.decsesclerosis lateral amiotrófica
dc.subject.decs/terapia
dc.subject.decsterapia genética
dc.subject.decsratones transgénicos
dc.relation.publishversionhttps://doi.org/10.3389/fneur.2021.693309
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.organismesInstitut Català de la Salut
dc.contributor.authoraffiliation[Mòdol-Caballero G, Herrando-Grabulosa M, Hernández N, Francos-Quijorna I] Departament de Biologia Cel•lular, Fisiologia i Immunologia, Institut de Neurociències, Universitat Autònoma de Barcelona, Bellaterra, Spain. Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Barcelona, Spain. [Verdés S] 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. [García-Lareu B] Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Barcelona, Spain. Departament de Bioquímica i Biologia Molecular, Institut de Neurociències, Universitat Autònoma de Barcelona, Bellaterra, Spain. [Bosch A] Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Barcelona, Spain. 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
dc.identifier.pmid34630277
dc.identifier.wos000703767700001
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


Files in this item

Thumbnail
Thumbnail
Thumbnail

This item appears in the following Collection(s)

Show simple item record