Base editing of haematopoietic stem cells rescues sickle cell disease in mice von Newby, Gregory A., Yen, Jonathan S., Woodard, Kaitly J., Mayuranathan, Thiyagaraj, Lazzarotto, Cicera R., Li, Yichao, Sheppard-Tillman, Heather, Porter, Shaina N., Yao, Yu, Mayberry, Kalin, Everette, Kelcee A., Jang, Yoonjeong, Podracky, Christopher J., Thaman, Elizabeth, Lechauve, Christophe, Sharma, Akshay, Henderson, Jordana M., Richter, Michelle F., Zhao, Kevin T., Miller, Shannon M., Wang, Tina, Koblan, Luke W., McCaffrey, Anton P., Tisdale, John F., Kalfa, Theodosia A., Pruett-Miller, Shondra M., Tsai, Shengdar Q., Weiss, Mitchell J., Liu, David R.

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Selective in vivo metabolic cell-labeling-mediated cancer targeting von Wang, Hua, Wang, Ruibo, Cai, Kaimin, He, Hua, Liu, Yang, Yen, Jonathan, Wang, Zhiyu, Xu, Ming, Sun, Yiwen, Zhou, Xin, Yin, Qian, Tang, Li, Dobrucki, Iwona T, Dobrucki, Lawrence W, Chaney, Eric J, Boppart, Stephen A, Fan, Timothy M, Lezmi, Stéphane, Chen, Xuesi, Yin, Lichen, Cheng, Jianjun

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Editing human hematopoietic stem cells: advances and challenges von Bhoopalan, Senthil Velan, Yen, Jonathan S., Levine, Rachel M., Sharma, Akshay

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Generation of induced pluripotent stem cells in the absence of drug selection von Blelloch, Robert, Venere, Monica, Yen, Jonathan, Ramalho-Santos, Miguel

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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells von Lian, Xizhen, Chatterjee, Sumanta, Sun, Yehui, Dilliard, Sean A., Moore, Stephen, Xiao, Yufen, Bian, Xiaoyan, Yamada, Kohki, Sung, Yun-Chieh, Levine, Rachel M., Mayberry, Kalin, John, Samuel, Liu, Xiaoye, Smith, Caroline, Johnson, Lindsay T., Wang, Xu, Zhang, Cheng Cheng, Liu, David R., Newby, Gregory A., Weiss, Mitchell J., Yen, Jonathan S., Siegwart, Daniel J.

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Potent and uniform fetal hemoglobin induction via base editing von Mayuranathan, Thiyagaraj, Newby, Gregory A., Feng, Ruopeng, Yao, Yu, Mayberry, Kalin D., Lazzarotto, Cicera R., Li, Yichao, Levine, Rachel M., Nimmagadda, Nikitha, Dempsey, Erin, Kang, Guolian, Porter, Shaina N., Doerfler, Phillip A., Zhang, Jingjing, Jang, Yoonjeong, Chen, Jingjing, Bell, Henry W., Crossley, Merlin, Bhoopalan, Senthil Velan, Sharma, Akshay, Tisdale, John F., Pruett-Miller, Shondra M., Cheng, Yong, Tsai, Shengdar Q., Liu, David R., Weiss, Mitchell J., Yen, Jonathan S.

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Preclinical development of lentiviral vector gene therapy for Diamond-Blackfan anemia syndrome von Bhoopalan, Senthil Velan, Mayuranathan, Thiyagaraj, Liu, Nana, Mayberry, Kalin, Yao, Yu, Zhang, Jingjing, Métais, Jean-Yves, Yan, Koon-Kiu, Throm, Robert E, Ellis, Steven R, Ju, Yan, Han, Lei, Suryaprakash, Shruthi, Palmer, Lance E, Zhou, Sheng, Yu, Jiyang, Cheng, Yong, Yen, Jonathan S, Gottschalk, Stephen, Weiss, Mitchell J

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Butyrate Greatly Enhances Derivation of Human Induced Pluripotent Stem Cells by Promoting Epigenetic Remodeling and the Expression of Pluripotency‐Associated Genes von Mali, Prashant, Chou, Bin‐Kuan, Yen, Jonathan, Ye, Zhaohui, Zou, Jizhong, Dowey, Sarah, Brodsky, Robert A., Ohm, Joyce E., Yu, Wayne, Baylin, Stephen B., Yusa, Kosuke, Bradley, Allan, Meyers, David J., Mukherjee, Chandrani, Cole, Philip A., Cheng, Linzhao

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease von Katta, Varun, O’Keefe, Kiera, Li, Yichao, Mayuranathan, Thiyagaraj, Lazzarotto, Cicera R., Wood, Rachael K., Levine, Rachel M., Powers, Alicia, Mayberry, Kalin, Manquen, Garret, Yao, Yu, Zhang, Jingjing, Jang, Yoonjeong, Nimmagadda, Nikitha, Dempsey, Erin A., Lee, GaHyun, Uchida, Naoya, Cheng, Yong, Fazio, Frank, Lockey, Tim, Meagher, Mike, Sharma, Akshay, Tisdale, John F., Zhou, Sheng, Yen, Jonathan S., Weiss, Mitchell J., Tsai, Shengdar Q.

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Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice von Everette, Kelcee A., Newby, Gregory A., Levine, Rachel M., Mayberry, Kalin, Jang, Yoonjeong, Mayuranathan, Thiyagaraj, Nimmagadda, Nikitha, Dempsey, Erin, Li, Yichao, Bhoopalan, Senthil Velan, Liu, Xiong, Davis, Jessie R., Nelson, Andrew T., Chen, Peter J., Sousa, Alexander A., Cheng, Yong, Tisdale, John F., Weiss, Mitchell J., Yen, Jonathan S., Liu, David R.

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TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells von Yen, Jonathan, Fiorino, Michael, Liu, Yi, Paula, Steve, Clarkson, Scott, Quinn, Lisa, Tschantz, William R., Klock, Heath, Guo, Ning, Russ, Carsten, Yu, Vionnie W. C., Mickanin, Craig, Stevenson, Susan C., Lee, Cameron, Yang, Yi

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Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations von Woodard, Kaitly J., Doerfler, Phillip A., Mayberry, Kalin D., Sharma, Akshay, Levine, Rachel, Yen, Jonathan, Valentine, Virginia, Palmer, Lance E., Valentine, Marc, Weiss, Mitchell J.

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Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells von Yen, Jonathan S, Newby, Gregory A., Mayuranathan, Thiyagaraj, Porter, Shaina N., Yao, Yu, Woodard, Kaitly J., Mayberry, Kalin, Everette, Kelcee, Zhang, Jingjing, Henderson, Jordana M, Mccaffrey, Anton P, Yen, Jonathan, Tisdale, John F., Tsai, Shengdar Q., Liu, David R., Weiss, Mitchell J.

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An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity von Bhoopalan, Senthil Velan, Yen, Jonathan S, Mayuranathan, Thiyagaraj, Mayberry, Kalin D, Yao, Yu, Lillo Osuna, Maria Angeles, Jang, Yoonjeong, Liyanage, Janaka Ss, Blanc, Lionel, Ellis, Steven R, Wlodarski, Marcin W, Weiss, Mitchell J

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Bioorthogonal Oxime Ligation Mediated In Vivo Cancer Targeting von Tang, Li, Yin, Qian, Xu, Yunxiang, Zhou, Qin, Cai, Kaimin, Yen, Jonathan, Dobrucki, Lawrence W, Cheng, Jianjun

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DEVELOPMENT OF NEW GENE EDITING APPROACHES FOR BONE MARROW FAILURE AND MDS PREDISPOSITION SYNDROMES von Krzyzanowski, Damian, Khiami, Mjad, Han, Lei, Sahoo, Sushree, Miller, Shondra, Tsai, Shengdar, Bhoopalan, Senthil, Yen, Jonathan, Wlodarski, Marcin

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Efficient Derivation and Genetic Modifications of Human Pluripotent Stem Cells on Engineered Human Feeder Cell Lines von Zou, Chunlin, Chou, Bin-Kuan, Dowey, Sarah N., Tsang, Kitman, Huang, Xiaosong, Liu, Cyndi F., Smith, Cory, Yen, Jonathan, Mali, Prashant, Zhang, Yu Alex, Cheng, Linzhao, Ye, Zhaohui

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Cationic, helical polypeptide-based gene delivery for IMR-90 fibroblasts and human embryonic stem cells von Yen, Jonathan, Zhang, Yanfeng, Gabrielson, Nathan P, Yin, Lichen, Guan, Linna, Chaudhury, Isthier, Lu, Hua, Wang, Fei, Cheng, Jianjun

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Cancer pain: Magdi Hanna, Zbigniew (Ben) Zylicz (editors). Springer-Verlag London, 2013, 286 pages, ebook version available. ISBN 978-0-85729-229-2 von Yen, Jonathan

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Directed evolution of adenine base editors with increased activity and therapeutic application von Gaudelli, Nicole M., Lam, Dieter K., Rees, Holly A., Solá-Esteves, Noris M., Barrera, Luis A., Born, David A., Edwards, Aaron, Gehrke, Jason M., Lee, Seung-Joo, Liquori, Alexander J., Murray, Ryan, Packer, Michael S., Rinaldi, Conrad, Slaymaker, Ian M., Yen, Jonathan, Young, Lauren E., Ciaramella, Giuseppe

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