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. 2020 Apr 15;11:494. doi: 10.3389/fimmu.2020.00494

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Copyright © 2020 Patel, Lundgren, Spencer and Doering.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

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Ex vivo CD68-ET3-LV CD34⁺ clinical gene therapy paradigm. Autologous CD34⁺ HSPC are isolated from subjects with hemophilia A, genetically modified ex vivo using LV encompassing a codon optimized pfVIII transgene (ET3) under the monocyte lineage restricted promoter, CD68. Genetically modified HSPCs are then infused back into the subject following non-myeloablative conditioning with immune suppression. Post-administration of the genetically-modified autologous cell product, plasma fVIII levels, vector copy number in peripheral blood, and fVIII immunity status are followed.