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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Detalhes bibliográficos

Principais autores:	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
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Frontiers Media S.A. 2026-01-01
Colecção:	Frontiers in Immunology
Assuntos:	hemophilia A FVIII gene therapy mesenchymal stromal cell transplantation placental cells
Acesso em linha:	https://www.frontiersin.org/articles/10.3389/fimmu.2025.1716950/full
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