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Showing 1–2 of 2 results
Advanced filters: Author: Selami Demirci Clear advanced filters

  • Successful engraftment of human hematopoietic stem cells during gene therapy requires myeloablative conditioning of the recipient, at the expense of toxicity. Authors show here that a single-dose of anti-CD117 antibody-drug conjugate achieves similar engraftment results as traditional multi-dose busulfan conditioning but preserves fertility in a non-human primate model.

    • Naoya Uchida
    • Ulana Stasula
    • John F. Tisdale
    ResearchOpen Access
    Nature Communications
    Volume: 14, P: 1-11

  • Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a forward orientation and show that it has improved titers and transduction efficiency in humanized mice and nonhuman primates.

    • Naoya Uchida
    • Matthew M. Hsieh
    • John F. Tisdale
    ResearchOpen Access
    Nature Communications
    Volume: 10, P: 1-14