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

Author

Listed:
  • 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))

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.

Suggested Citation

  • 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 &, 2023. "Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity," Nature Communications, Nature, vol. 14(1), pages 1-17, December.
    • Handle: RePEc:nat:natcom:v:14:y:2023:i:1:d:10.1038_s41467-023-39986-1
    DOI: 10.1038/s41467-023-39986-1
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    References listed on IDEAS

    as
    1. Sourav K. Bose & Brandon M. White & Meghana V. Kashyap & Apeksha Dave & Felix R. De Bie & Haiying Li & Kshitiz Singh & Pallavi Menon & Tiankun Wang & Shiva Teerdhala & Vishal Swaminathan & Heather A. , 2021. "In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease," Nature Communications, Nature, vol. 12(1), pages 1-16, December.
    2. Brian C. Searle & Kristian E. Swearingen & Christopher A. Barnes & Tobias Schmidt & Siegfried Gessulat & Bernhard Küster & Mathias Wilhelm, 2020. "Generating high quality libraries for DIA MS with empirically corrected peptide predictions," Nature Communications, Nature, vol. 11(1), pages 1-10, December.
    Full references (including those not matched with items on IDEAS)

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