Contact

  • Academic
    fekman@stanford.edu
    University - Student Department: Genetics Position: Graduate
    University - Student Department: School of Medicine Position: Graduate, Medicine

Additional Info

All Publications

  • Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis. Nature communications Shah, A. P., Majeti, K. R., Ekman, F. K., Selvaraj, S., Sharma, D., Sinha, R., Soupene, E., Chati, P., Luna, S. E., Charlesworth, C. T., McCreary, T., Lesch, B. J., Tran, T., Chu, S. N., Porteus, M. H., Kyle Cromer, M. 2025; 16 (1): 1140

    Abstract

    Blood transfusion plays a vital role in modern medicine, but frequent shortages occur. Ex vivo manufacturing of red blood cells (RBCs) from universal donor cells offers a potential solution, yet the high cost of recombinant cytokines remains a barrier. Erythropoietin (EPO) signaling is crucial for RBC development, and EPO is among the most expensive media components. To address this challenge, we develop highly optimized small molecule-inducible synthetic EPO receptors (synEPORs) using design-build-test cycles and genome editing. By integrating synEPOR at the endogenous EPOR locus in O-negative induced pluripotent stem cells, we achieve equivalent erythroid differentiation, transcriptomic changes, and hemoglobin production using small molecules compared to EPO-supplemented cultures. This approach dramatically reduces culture media costs. Our strategy not only addresses RBC production challenges but also demonstrates how protein and genome engineering can introduce precisely regulated cellular behaviors, potentially improving scalable manufacturing of a wide range of clinically relevant cell types.

    View details for DOI 10.1038/s41467-025-56239-5

    View details for PubMedID 39880867

    View details for PubMedCentralID 5355882

  • Targeting the <i>JAK2</i>-V617F Mutation in Polycythemia Vera Using CRISPR/AAV6 Genome Editing Ekman, F., Selvaraj, S., Gotlib, J., Cromer, M., Porteus, M. H. ELSEVIER. 2024: 4524-4525

    View details for DOI 10.1182/blood-2024-211120

    View details for Web of Science ID 001412530900020

  • High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition. Nature biotechnology Selvaraj, S., Feist, W. N., Viel, S., Vaidyanathan, S., Dudek, A. M., Gastou, M., Rockwood, S. J., Ekman, F. K., Oseghale, A. R., Xu, L., Pavel-Dinu, M., Luna, S. E., Cromer, M. K., Sayana, R., Gomez-Ospina, N., Porteus, M. H. 2023

    Abstract

    Therapeutic applications of nuclease-based genome editing would benefit from improved methods for transgene integration via homology-directed repair (HDR). To improve HDR efficiency, we screened six small-molecule inhibitors of DNA-dependent protein kinase catalytic subunit (DNA-PKcs), a key protein in the alternative repair pathway of non-homologous end joining (NHEJ), which generates genomic insertions/deletions (INDELs). From this screen, we identified AZD7648 as the most potent compound. The use of AZD7648 significantly increased HDR (up to 50-fold) and concomitantly decreased INDELs across different genomic loci in various therapeutically relevant primary human cell types. In all cases, the ratio of HDR to INDELs markedly increased, and, in certain situations, INDEL-free high-frequency (>50%) targeted integration was achieved. This approach has the potential to improve the therapeutic efficacy of cell-based therapies and broaden the use of targeted integration as a research tool.

    View details for DOI 10.1038/s41587-023-01888-4

    View details for PubMedID 37537500

    View details for PubMedCentralID 3694601

https://orcid.org/0000-0003-3935-4685