Serrano, Luis J.De la Torre, PazLiras, AntonioFlores, Ana I.2023-06-162023-06-162021-07-280753-332210.1016/j.biopha.2021.112059https://hdl.handle.net/20.500.14352/4688CRUE-CSIC (Acuerdos Transformativos 2021)Deficiency 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.engAtribución-NoComercial-SinDerivadas 3.0 Españajournal articlehttps://doi.org/10.1016/j.biopha.2021.112059open access616.151.5615.361.018.1Inherited coagulopathiesSevere human factor VDeficiencyCell therapyHuman deciduaMesenchymal stem cellsHepatocytesHematologíaBiotecnología3205.04 Hematología3399 Otras Especialidades Tecnológicas