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



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Serrano, Luis J. and De la Torre, Paz and Liras, Antonio and Flores, Ana I. (2021) Cell therapy for factor V deficiency: an approach based on human decidua mesenchymal stem cells. Biomedicine & pharmacotherapy, 142 (112059). pp. 1-8. ISSN 0753-3322

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Official URL: https://doi.org/10.1016/j.biopha.2021.112059


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.

Item Type:Article
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CRUE-CSIC (Acuerdos Transformativos 2021)

Uncontrolled Keywords:Inherited coagulopathies; Severe human factor V; Deficiency; Cell therapy; Human decidua; Mesenchymal stem cells; Hepatocytes
Subjects:Medical sciences > Medicine > Hematology
Medical sciences > Biology > Biotechnology
ID Code:70213
Deposited On:09 Feb 2022 09:03
Last Modified:21 Feb 2022 11:18

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