Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity

Martin Rodriguez, Brady Trevisan, Ritu M. Ramamurthy, Sunil K. George, Jonathan Diaz, Jordan Alexander, Diane Meares, Denise J. Schwahn, David R. Quilici, Jorge Figueroa, Michael Gautreaux, Andrew Farland, Anthony Atala, Christopher B. Doering, H. Trent Spencer, Christopher D. Porada and Graça Almeida-Porada ()
Additional contact information
Martin Rodriguez: Wake Forest School of Medicine (WFSOM)
Brady Trevisan: Wake Forest School of Medicine (WFSOM)
Ritu M. Ramamurthy: Wake Forest School of Medicine (WFSOM)
Sunil K. George: Wake Forest School of Medicine (WFSOM)
Jonathan Diaz: Wake Forest School of Medicine (WFSOM)
Jordan Alexander: Emory University
Diane Meares: Wake Forest School of Medicine
Denise J. Schwahn: Wave Life Sciences
David R. Quilici: University of Nevada Reno
Jorge Figueroa: Center for Research in Obstetrics and Gynecology, WFSOM
Michael Gautreaux: HLA/Immunogenetics and Immunodiagnostics Laboratories
Andrew Farland: Wake Forest School of Medicine
Anthony Atala: Wake Forest School of Medicine (WFSOM)
Christopher B. Doering: Emory University
H. Trent Spencer: Emory University
Christopher D. Porada: Wake Forest School of Medicine (WFSOM)
Graça Almeida-Porada: Wake Forest School of Medicine (WFSOM)

Nature Communications, 2023, vol. 14, issue 1, 1-17

Abstract: Abstract Hemophilia A is the most common X-linked bleeding disorder affecting more than half-a-million individuals worldwide. Persons with severe hemophilia A have coagulation FVIII levels 3 years post-treatment. Cells engraft in major organs, and none of the recipients mount immune responses to either the cells or the FVIII they produce. Thus, these studies attest to the feasibility, immunologic advantage, and safety of treating hemophilia A prior to birth.

Date: 2023
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DOI: 10.1038/s41467-023-39986-1

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