Transplanted gene-modified placental cells boost FVIII activity in pediatric sheep without eliciting immunity, toxicity, or adverse events
Data show that while intravenous (IV) infusion of ET3 or hFVIII to pediatric sheep results in a high level of inhibitory antibodies, administration of PLC-mcoET3 IV is safe, and resulted in increased plasma FVIII activity for at least 15 weeks without the formation of anti-ET3/FVIII inhibitory antibodies.
Authors
- Michael D. Gautreaux (ORCID: https://orcid.org/0000-0003-3666-7010)
- Jacqueline Dizon
- Christopher D. Porada (ORCID: https://orcid.org/0000-0002-7321-7556)
- Brady Trevisan
- Denise J. Schwahn (ORCID: https://orcid.org/0000-0001-8665-2402)
- Christopher B. Doering (ORCID: https://orcid.org/0000-0002-5662-0060)
- Martin Rodriguez (ORCID: https://orcid.org/0000-0003-4493-0130)
- Anthony Atala (ORCID: https://orcid.org/0000-0001-8186-2160)
- Andrew M. Farland
- Shannon Lankford
- Ritu M. Ramamurthy (ORCID: https://orcid.org/0000-0002-7131-6702)
- Oluwaseun Babatunde
- John J. T. Owen (ORCID: https://orcid.org/0000-0001-8882-3340)
- Almeida-Porada Graca
- Sunil George
- Jordan Shields
- H. Trent Spencer
Institutions
- Zoetis (United States) (US)
- Children's Healthcare of Atlanta (US)
- Wake Forest University (US)
Publication Details
- Journal
- Frontiers in Immunology
- Published
- 2026-01-07
- DOI
- https://doi.org/10.3389/fimmu.2025.1716950
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00