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Cell therapy for factor V deficiency: an approach based on human decidua mesenchymal stem cells

dc.contributor.authorSerrano, Luis
dc.contributor.authorDe la Torre, Paz
dc.contributor.authorLiras, Antonio
dc.contributor.authorFlores, Ana
dc.date.accessioned2023-06-16T14:19:34Z
dc.date.available2023-06-16T14:19:34Z
dc.date.issued2021
dc.descriptionCRUE-CSIC (Acuerdos Transformativos 2021)
dc.description.abstractDeficiency of factor V is a congenital autosomal recessive coagulopathy associated with mutations in the F5 gene that results in mild-to-severe bleeding episodes. Factor V is a component of the prothrombinase complex responsible for accelerating conversion of prothrombin to thrombin. At the present time there are no therapeutic factor V concentrates available. This study was designed to lay the preliminary foundations for future cell-based therapy for patients with severe factor V deficiency. The study showed that hepatospheres, which produce coagulation factors VIII, IX, and V, synthetize and store intracellular glycogen and express albumin levels up to 8 times higher than those of undifferentiated cells. Factor IX and factor V gene expression increased significantly in hepatospheres as compared to undifferentiated cells, whereas factor VIII gene expression remained constant. The factor V protein was detected in the hepatospheres´ secretome. Considering the enormous potential of mesenchymal stem cells as therapeutic agents, this study proposes a highly reproducible method to induce differentiation of mesenchymal stem cells from human placenta to factor V-producing hepatospheres. This strategy constitutes a preliminary step towards a curative treatment of factor V deficiency through advanced therapies such as cell therapy.
dc.description.departmentDepto. de Genética, Fisiología y Microbiología
dc.description.facultyFac. de Ciencias Biológicas
dc.description.refereedTRUE
dc.description.sponsorshipMinisterio de Economía y Competitividad (MINECO)/Instituto de Salud Carlos III
dc.description.sponsorshipComunidad de Madrid/FEDER
dc.description.sponsorshipAsociación Andaluza de Hemofilia/Octapharma S.A
dc.description.sponsorshipSoria Melguizo Foundation
dc.description.statuspub
dc.eprint.idhttps://eprints.ucm.es/id/eprint/70213
dc.identifier.doi10.1016/j.biopha.2021.112059
dc.identifier.issn0753-3322
dc.identifier.officialurlhttps://doi.org/10.1016/j.biopha.2021.112059
dc.identifier.urihttps://hdl.handle.net/20.500.14352/4688
dc.issue.number112059
dc.journal.titleBiomedicine & pharmacotherapy
dc.language.isoeng
dc.page.final8
dc.page.initial1
dc.publisherElsevier
dc.relation.projectID(PI15/01803)
dc.relation.projectID(S2013/MIT-2862-CM)
dc.relation.projectID(ASANHEMO FV2016–20)
dc.relation.projectID(PI2013/0116)
dc.rightsAtribución-NoComercial-SinDerivadas 3.0 España
dc.rights.accessRightsopen access
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/3.0/es/
dc.subject.cdu616.151.5
dc.subject.cdu615.361.018.1
dc.subject.keywordInherited coagulopathies
dc.subject.keywordSevere human factor V
dc.subject.keywordDeficiency
dc.subject.keywordCell therapy
dc.subject.keywordHuman decidua
dc.subject.keywordMesenchymal stem cells
dc.subject.keywordHepatocytes
dc.subject.ucmHematología
dc.subject.ucmBiotecnología
dc.subject.unesco3205.04 Hematología
dc.subject.unesco3399 Otras Especialidades Tecnológicas
dc.titleCell therapy for factor V deficiency: an approach based on human decidua mesenchymal stem cells
dc.typejournal article
dc.volume.number142
dspace.entity.typePublication

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