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CD117 Antibody Conditioning and Multiplex Base Editing Enable Rapid and Robust Fetal Hemoglobin Reactivation in a Rhesus Autologous Transplantation Model CD117抗体预处理与多重碱基编辑技术实现灵长类自体移植模型中快速且稳定的胎儿血红蛋白重激活 Demirci, Selami; Mondal, Nandini; Austin, Wayne; Butt, Henna; London, Evan; Sathish, Shruti; Zhang, Kathy; Wong, Jeffrey; Kromer, Hugh; Harmon, Alexander; Decker, Jeremy; Le, Anh; Essawi, Khaled; Krouse, Allen E.; Linde, N. Seth; Bonifacino, Aylin; Engels, Theresa; Golomb, Justin; Hong, So Gun; Uchida, Naoya; Gaudelli, Nicole; Hartigan, Adam J.; Mcdonagh, Charlotte F.; Chu, S. Haihua; Donahue, Robert E.; Tisdale, John 分享 收藏
Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models 离体培养静息时间影响基因组编辑的人类造血干细胞和祖细胞在异种移植小鼠模型中的移植结果 Demirci, Selami; Khan, Muhammad B. N.; Hinojosa, Gabriela; Le, Anh; Leonard, Alexis; Essawi, Khaled; Gudmundsdottir, Bjorg; Liu, Xiong; Zeng, Jing; Inam, Zaina; Chu, Rebecca; Uchida, Naoya; Araki, Daisuke; London, Evan; Butt, Henna; Bauer, Stacy A. Maitland ' Daniel E.; Wolfe, Scot A.; Larochelle, Andre; Tisdale, John F. 分享 收藏
In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation Leonard, Alexis K.; Furstenau, Dana; Inam, Zaina; Luckett, Christina; Chu, Rebecca; Demirci, Selami; Essawi, Khaled; Gudmundsdottir, Bjorg; Hinds, Malikiya; DiNicola, Julia; Li, Quan; Eaton, William A.; Cellmer, Troy; Wang, Xunde; Thein, Swee Lay; Macari, Elizabeth R.; VanNest, Sara; Hsieh, Matthew M.; Bonner, Melissa; Pierciey, Francis J.; Tisdale, John F. 分享 收藏
Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model 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. 分享 收藏
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Forced enhancer-promoter rewiring to alter in animal models Peslak, Scott A.; Demirci, Selami; Chandra, Vemika; Ryu, Byoung; Bhardwaj, Saurabh K.; Jiang, Jing; Rupon, Jeremy W.; Throm, Robert E.; Uchida, Naoya; Leonard, Alexis; Essawi, Khaled; Bonifacino, Aylin C.; Krouse, Allen E.; Linde, Nathaniel S.; Donahue, Robert E.; Ferrara, Francesca; Wielgosz, Matthew; Abdulmalik, Osheiza; Hamagami, Nicole; Germino-Watnick, Paula; Le, Anh; Chu, Rebecca; Hinds, Malikiya; Weiss, Mitchell J.; Tong, Wei; Tisdale, John F.; Blobel, Gerd A. 分享 收藏
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Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor Uchida, Naoya; Ferrara, Francesca; Drysdale, Claire M.; Yapundich, Morgan; Gamer, Jackson; Nassehi, Tina; DiNicola, Julia; Shibata, Yoshitaka; Wielgosz, Matthew; Kim, Yoon-Sang; Bauler, Matthew; Throm, Robert E.; Haro-Mora, Juan J.; Demirci, Selami; Bonifacino, Aylin C.; Krouse, Allen E.; Linde, N. Seth; Donahue, Robert E.; Ryu, Byoung; Tisdale, John F. 分享 收藏
Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models Uchida, Naoya; Li, Linhong; Nassehi, Tina; Drysdale, Claire M.; Yapundich, Morgan; Gamer, Jackson; Haro-Mora, Juan J.; Demirci, Selami; Leonard, Alexis; Bonifacino, Aylin C.; Krouse, Allen E.; Linde, N. Seth; Allen, Cornell; Peshwa, Madhusudan, V; De Ravin, Suk See; Donahue, Robert E.; Malech, Harry L.; Tisdale, John F. 分享 收藏
Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation (vol 11, 493, 2020) Demirci, Selami; Haro-Mora, Juan J.; Leonard, Alexis; Drysdale, Claire; Malide, Daniela; Keyvanfar, Keyvan; Essawi, Khaled; Vizcardo, Raul; Tamaoki, Naritaka; Restifo, Nicholas P.; Tisdale, John F.; Uchida, Naoya 分享 收藏
Definitive hematopoietic stem/progenitor cells from human embryonic stem cells through serum/feeder-free organoid-induced differentiation Demirci, Selami; Haro-Mora, Juan J.; Leonard, Alexis; Drysdale, Claire; Malide, Daniela; Keyvanfar, Keyvan; Essawi, Khaled; Vizcardo, Raul; Tamaoki, Naritaka; Restifo, Nicholas P.; Tisdale, John F.; Uchida, Naoya 分享 收藏
BCL11A enhancer edited hematopoietic stem cells persist in rhesus monkeys without toxicity BCL11A增强子编辑的造血干细胞在恒河猴中持续存在而没有毒性 Demirci, Selami; Zeng, Jing; Wu, Yuxuan; Uchida, Naoya; Shen, Anne H.; Pellin, Danilo; Gamer, Jackson; Yapundich, Morgan; Drysdale, Claire; Bonanno, Jasmine; Bonifacino, Aylin C.; Krouse, Allen E.; Linde, Nathaniel S.; Engels, Theresa; Donahue, Robert E.; Haro-Mora, Juan J.; Leonard, Alexis; Nassehi, Tina; Luk, Kevin; Porter, Shaina N.; Lazzarotto, Cicera R.; Tsai, Shengdar Q.; Weiss, Mitchell J.; Pruett-Miller, Shondra M.; Wolfe, Scot A.; Bauer, Daniel E.; Tisdale, John F. 分享 收藏
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Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders 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. 分享 收藏
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