2026-08-20T15:39:41Zhttps://docta.ucm.es/rest/oai/request

oai:docta.ucm.es:20.500.14352/46882025-02-13T17:14:20Zcom_20.500.14352_14col_20.500.14352_15

Cell therapy for factor V deficiency: an approach based on human decidua mesenchymal stem cells Serrano, Luis De la Torre, Paz Liras, Antonio Flores, Ana 616.151.5 615.361.018.1 Inherited coagulopathies Severe human factor V Deficiency Cell therapy Human decidua Mesenchymal stem cells Hepatocytes Hematología Biotecnología 3205.04 Hematología 3399 Otras Especialidades Tecnológicas CRUE-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. Ministerio de Economía y Competitividad (MINECO)/Instituto de Salud Carlos III Comunidad de Madrid/FEDER Asociación Andaluza de Hemofilia/Octapharma S.A Soria Melguizo Foundation Depto. de Genética, Fisiología y Microbiología Fac. de Ciencias Biológicas TRUE pub 2023-06-16T14:19:34Z 2023-06-16T14:19:34Z 2021 journal article https://hdl.handle.net/20.500.14352/4688 0753-3322 10.1016/j.biopha.2021.112059 eng (PI15/01803) (S2013/MIT-2862-CM) (ASANHEMO FV2016–20) (PI2013/0116) Atribución-NoComercial-SinDerivadas 3.0 España https://creativecommons.org/licenses/by-nc-nd/3.0/es/ open access application/pdf Elsevier