Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice by Alexis Leonard, Morgan Yapundich, Tina Nassehi, Jackson Gamer, Claire M. Drysdale, Juan J. Haro-Mora, Selami Demirci, Matthew M. Hsieh, Naoya Uchida, John F. Tisdale

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βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells by Selami Demirci, Bjorg Gudmundsdottir, Quan Li, Juan J. Haro-Mora, Tina Nassehi, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Fayaz Seifuddin, John F. Tisdale, Naoya Uchida

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Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease by Naoya Uchida, Claire M. Drysdale, Tina Nassehi, Jackson Gamer, Morgan Yapundich, Julia DiNicola, Yoshitaka Shibata, Malikiya Hinds, Bjorg Gudmundsdottir, Juan J. Haro-Mora, Selami Demirci, John F. Tisdale

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Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models by Naoya Uchida, Linhong Li, Tina Nassehi, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Juan J. Haro-Mora, Selami Demirci, Alexis Leonard, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Cornell Allen, Madhusudan V. Peshwa, Suk See De Ravin, Robert E. Donahue, Harry L. Malech, John F. Tisdale

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