Autologous bone marrow-derived MSCs engineered to express oFVIII-FLAG engraft in adult sheep and produce an effective increase in plasma FVIII levels

Autologous bone marrow-derived MSCs engineered to express oFVIII-FLAG engraft in adult sheep and produce an effective increase in plasma FVIII levels

IntroductionHemophilia A (HA) is the most common X-linked bleeding disorder, occurring in 1 in 5,000 live male births and affecting >1 million individuals worldwide. Although advances in protein-based HA therapeutics have improved health outcomes, current standard-of-care requires infusion 2-...

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Bibliographic Details
Main Authors: Brady Trevisan, Martin Rodriguez, Hailey Medder, Shannon Lankford, Rebecca Combs, John Owen, Anthony Atala, Christopher D. Porada, Graça Almeida-Porada
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-12-01
Series:Frontiers in Immunology
Subjects:
mesenchymal stroma cell
cell therapy
bone marrow
gene therapy
Hemophilia A
efficacy & safety
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2022.1070476/full

Internet

https://www.frontiersin.org/articles/10.3389/fimmu.2022.1070476/full

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