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Transplanted gene-modified placental cells boost FVIII activity in pediatric sheep without eliciting immunity, toxicity, or adverse events

BackgroundThe current standard of care for Hemophilia A (HA), a hereditary bleeding disorder caused by mutations in the Factor VIII (F8) gene, include FVIII replacement proteins, engineered clotting factors, and a broad array of new therapeutics including antibodies and gene therapy. These therapies...

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Huvudupphov: Brady Trevisan, Martin Rodriguez, Ritu Ramamurthy, Sunil George, Oluwaseun O. Babatunde, Jacqueline Dizon, Jordan Shields, Shannon Lankford, Denise Schwahn, Michael Gautreaux, Andrew Farland, John Owen, Anthony Atala, Christopher B. Doering, H. Trent Spencer, Christopher D. Porada, Graça Almeida-Porada
Materialtyp: Artigo
Språk:Inglês
Utgiven: Frontiers Media S.A. 2026-01-01
Serie:Frontiers in Immunology
Ämnen:
hemophilia A
FVIII
gene therapy
mesenchymal stromal cell
transplantation
placental cells
Länkar:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1716950/full
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