Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells von Cromer, M. Kyle, Camarena, Joab, Martin, Renata M., Lesch, Benjamin J., Vakulskas, Christopher A., Bode, Nicole M., Kurgan, Gavin, Collingwood, Michael A., Rettig, Garrett R., Behlke, Mark A., Lemgart, Viktor T., Zhang, Yankai, Goyal, Ankush, Zhao, Feifei, Ponce, Ezequiel, Srifa, Waracharee, Bak, Rasmus O., Uchida, Naoya, Majeti, Ravindra, Sheehan, Vivien A., Tisdale, John F., Dever, Daniel P., Porteus, Matthew H.

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Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study von Kanter, Julie, Thompson, Alexis A., Pierciey, Francis J., Hsieh, Matthew, Uchida, Naoya, Leboulch, Philippe, Schmidt, Manfred, Bonner, Melissa, Guo, Ruiting, Miller, Alex, Ribeil, Jean‐Antoine, Davidson, David, Asmal, Mohammed, Walters, Mark C., Tisdale, John F.

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Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor von Uchida, Naoya, Leonard, Alexis, Stroncek, David, Panch, Sandhya R, West, Kamille, Molloy, Eoghan, Hughes, Thomas E, Hauffe, Sara, Taylor, Tiffani, Fitzhugh, Courtney, Hankins, Jane S, Wilson, Megan, Sharma, Akshay, Tsai, Shengdar Q, Weiss, Mitch J, Hsieh, Matthew, Tisdale, John F

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Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model von Uchida, Naoya, Stasula, Ulana, Demirci, Selami, Germino-Watnick, Paula, Hinds, Malikiya, Le, Anh, Chu, Rebecca, Berg, Alexander, Liu, Xiong, Su, Ling, Wu, Xiaolin, Krouse, Allen E., Linde, N. Seth, Bonifacino, Aylin, Hong, So Gun, Dunbar, Cynthia E., Lanieri, Leanne, Bhat, Anjali, Palchaudhuri, Rahul, Bennet, Bindu, Hoban, Megan, Bertelsen, Kirk, Olson, Lisa M., Donahue, Robert E., Tisdale, John F.

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Evaluation of genetic diversity using 31 microsatellites in Miyako horses von MASUDA, Mioko, NIKADORI, Akihiro, NIKADORI, Emi, UCHIDA, Naoya, TAKIZAWA, Yoshimasa, ISHIMARU, Mutsuki, YAMAMOTO, Takumi, YUASA, Reiko, KUGAI, Haruo, NAGAHAMA, Yukio, TAKASU, Masaki, TOZAKI, Teruaki

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Prostaglandin E2 Enhances Human Cord Blood Stem Cell Xenotransplants and Shows Long-Term Safety in Preclinical Nonhuman Primate Transplant Models von Goessling, Wolfram, Allen, Robyn S., Guan, Xiao, Jin, Ping, Uchida, Naoya, Dovey, Michael, Harris, James M., Metzger, Mark E., Bonifacino, Aylin C., Stroncek, David, Stegner, Joseph, Armant, Myriam, Schlaeger, Thorsten, Tisdale, John F., Zon, Leonard I., Donahue, Robert E., North, Trista E.

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Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders von Uchida, Naoya, Hsieh, Matthew M., Raines, Lydia, Haro-Mora, Juan J., Demirci, Selami, Bonifacino, Aylin C., Krouse, Allen E., Metzger, Mark E., Donahue, Robert E., Tisdale, John F.

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Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease von Germino-Watnick, Paula, Hinds, Malikiya, Le, Anh, Chu, Rebecca, Liu, Xiong, Uchida, Naoya

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Genome editing of HBG1 and HBG2 to induce fetal hemoglobin von Métais, Jean-Yves, Doerfler, Phillip A., Mayuranathan, Thiyagaraj, Bauer, Daniel E., Fowler, Stephanie C., Hsieh, Matthew M., Katta, Varun, Keriwala, Sagar, Lazzarotto, Cicera R., Luk, Kevin, Neel, Michael D., Perry, S. Scott, Peters, Samuel T., Porter, Shaina N., Ryu, Byoung Y., Sharma, Akshay, Shea, Devlin, Tisdale, John F., Uchida, Naoya, Wolfe, Scot A., Woodard, Kaitly J., Wu, Yuxuan, Yao, Yu, Zeng, Jing, Pruett-Miller, Shondra, Tsai, Shengdar Q., Weiss, Mitchell J.

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Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease von Lessard, Samuel, Rimmelé, Pauline, Ling, Hui, Moran, Kevin, Vieira, Benjamin, Lin, Yi-Dong, Rajani, Gaurav Manohar, Hong, Vu, Reik, Andreas, Boismenu, Richard, Hsu, Ben, Chen, Michael, Cockroft, Bettina M., Uchida, Naoya, Tisdale, John, Alavi, Asif, Krishnamurti, Lakshmanan, Abedi, Mehrdad, Galeon, Isobelle, Reiner, David, Wang, Lin, Ramezi, Anne, Rendo, Pablo, Walters, Mark C., Levasseur, Dana, Peters, Robert, Harris, Timothy, Hicks, Alexandra

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

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Biallelic correction of sickle cell disease‐derived induced pluripotent stem cells (iPSCs) confirmed at the protein level through serum‐free iPS‐sac/erythroid differentiation von Haro‐Mora, Juan J., Uchida, Naoya, Demirci, Selami, Wang, Qi, Zou, Jizhong, Tisdale, John F.

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Robust generation of erythroid and multilineage hematopoietic progenitors from human iPSCs using a scalable monolayer culture system von Ruiz, Juan Pablo, Chen, Guibin, Haro Mora, Juan Jesus, Keyvanfar, Keyvan, Liu, Chengyu, Zou, Jizhong, Beers, Jeanette, Bloomer, Hanan, Qanash, Husam, Uchida, Naoya, Tisdale, John F., Boehm, Manfred, Larochelle, Andre

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Path to the Clinic: Assessment of iPSC-Based Cell Therapies In Vivo in a Nonhuman Primate Model von Hong, So Gun, Winkler, Thomas, Wu, Chuanfeng, Guo, Vicky, Pittaluga, Stefania, Nicolae, Alina, Donahue, Robert E., Metzger, Mark E., Price, Sandra D., Uchida, Naoya, Kuznetsov, Sergei A., Kilts, Tina, Li, Li, Robey, Pamela G., Dunbar, Cynthia E.

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Immunoresponse to Gene-Modified Hematopoietic Stem Cells von Drysdale, Claire M., Tisdale, John F., Uchida, Naoya

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Hematopoietic Stem Cell-Targeted Gene Therapy Using Lentiviral Vectors von Uchida, Naoya

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High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2 von Uchida, Naoya, Nassehi, Tina, Drysdale, Claire M., Gamer, Jackson, Yapundich, Morgan, Demirci, Selami, Haro-Mora, Juan J., Leonard, Alexis, Hsieh, Matthew M., Tisdale, John F.

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

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

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MASL1 induces erythroid differentiation in human erythropoietin-dependent CD34+ cells through the Raf/MEK/ERK pathway von Kumkhaek, Chutima, Aerbajinai, Wulin, Liu, Wenli, Zhu, Jianqiong, Uchida, Naoya, Kurlander, Roger, Hsieh, Matthew M., Tisdale, John F., Rodgers, Griffin P.

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