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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主要な著者:	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
フォーマット:	Artigo
言語:	Inglês
出版事項:	Frontiers Media S.A. 2026-01-01
シリーズ:	Frontiers in Immunology
主題:	hemophilia A FVIII gene therapy mesenchymal stromal cell transplantation placental cells
オンライン･アクセス:	https://www.frontiersin.org/articles/10.3389/fimmu.2025.1716950/full
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