BETA: Related publications

Publications

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Castiello MC, Brandas C, Ferrari S, et al. (2024) Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency. Science Translational Medicine. 16: eadh8162
Canarutto D, Asperti C, Vavassori V, et al. (2023) Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4 T-cells for the treatment of Hyper IgM1. The Embo Journal. e114188
Kerzel T, Giacca G, Beretta S, et al. (2023) In vivo macrophage engineering reshapes the tumor microenvironment leading to eradication of liver metastases. Cancer Cell. 41: 1892-1910.e10
Asperti C, Canarutto D, Porcellini S, et al. (2023) Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated and . Molecular Therapy. Methods & Clinical Development. 30: 546-557
Ferrari S, Naldini L. (2023) A step toward stem cell engineering in vivo. Science (New York, N.Y.). 381: 378-379
Canarutto D, Omer Javed A, Pedrazzani G, et al. (2023) Mobilization-based engraftment of haematopoietic stem cells: a new perspective for chemotherapy-free gene therapy and transplantation. British Medical Bulletin
Vavassori V, Ferrari S, Beretta S, et al. (2023) Lipid Nanoparticles Allow Efficient and Harmless Ex Vivo Gene Editing of Human Hematopoietic Cells. Blood
Ferrari S, Valeri E, Conti A, et al. (2023) Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy. Cell Stem Cell. 30: 549-570
Omer-Javed A, Pedrazzani G, Albano L, et al. (2022) Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells. Cell. 185: 2248-2264.e21
Milani M, Canepari C, Liu T, et al. (2022) Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates. Nature Communications. 13: 2454
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