Search Results - "Yen, Jonathan"

Base editing of haematopoietic stem cells rescues sickle cell disease in mice by 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.

“…Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB 1 . We used a custom adenine base editor (ABE8e-NRCH) 2 , 3 to convert the SCD…”
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Selective in vivo metabolic cell-labeling-mediated cancer targeting by 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

“…Metabolic labeling of the cell surface with a caged azide sugar enabled cleavage-mediated activation by enzymes overexpressed in cancer cells, allowing…”
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Editing human hematopoietic stem cells: advances and challenges by Bhoopalan, Senthil Velan, Yen, Jonathan S., Levine, Rachel M., Sharma, Akshay

“…Genome editing of hematopoietic stem and progenitor cells is being developed for the treatment of several inherited disorders of the hematopoietic system. The…”
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Generation of induced pluripotent stem cells in the absence of drug selection by Blelloch, Robert, Venere, Monica, Yen, Jonathan, Ramalho-Santos, Miguel

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Potent and uniform fetal hemoglobin induction via base editing by 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.

“…Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle cell disease. We compared five strategies in CD34 + hematopoietic…”
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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells by 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.

“…Therapeutic genome editing of haematopoietic stem cells (HSCs) would provide long-lasting treatments for multiple diseases. However, the in vivo delivery of…”
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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 by 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

“…We report here that butyrate, a naturally occurring fatty acid commonly used as a nutritional supplement and differentiation agent, greatly enhances the…”
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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 by 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

“…Sickle cell disease (SCD) is a common, severe genetic blood disorder. Current pharmacotherapies are partially effective and allogeneic hematopoietic stem cell…”
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Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice by 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.

“…Sickle-cell disease (SCD) is caused by an A·T-to-T·A transversion mutation in the β -globin gene ( HBB ). Here we show that prime editing can correct the SCD…”
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TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells by 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

“…CRISPR/Cas9 mediated gene editing of patient-derived hematopoietic stem and progenitor cells (HSPCs) ex vivo followed by autologous transplantation of the…”
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Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations by 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.

“…ABSTRACT We characterized the human β-like globin transgenes in two mouse models of sickle cell disease (SCD) and tested a genome-editing strategy to induce…”
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Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells by 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.

“…Sickle cell disease (SCD) is a chronic, life-altering multisystem disorder that affects millions of individuals worldwide. Strategies for genetic therapy of…”
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Bioorthogonal Oxime Ligation Mediated In Vivo Cancer Targeting by Tang, Li, Yin, Qian, Xu, Yunxiang, Zhou, Qin, Cai, Kaimin, Yen, Jonathan, Dobrucki, Lawrence W, Cheng, Jianjun

“…Current cancer targeting relying on specific biological interaction between cell surface antigen and respective antibody or its analogue has proven to be…”
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An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity by 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

“…Diamond-Blackfan anemia (DBA) is a genetic blood disease caused by heterozygous loss-of-function mutations in ribosomal protein (RP) genes, most commonly…”
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DEVELOPMENT OF NEW GENE EDITING APPROACHES FOR BONE MARROW FAILURE AND MDS PREDISPOSITION SYNDROMES by 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 by 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

“…Derivation of pluripotent stem cells (iPSCs) induced from somatic cell types and the subsequent genetic modifications of disease-specific or patient-specific…”
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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 by Yen, Jonathan

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Treating pain associated with head and neck cancer by WILLIAMS, John-E, YEN, Jonathan-Tc

“…Treatment of head and neck cancer pain by classifying pain into 4 categories and identifying special pain syndromes. The 4 categories are described and the…”
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Directed evolution of adenine base editors with increased activity and therapeutic application by 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

“…The foundational adenine base editors (for example, ABE7.10) enable programmable A•T to G•C point mutations but editing efficiencies can be low at challenging…”
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Gene Therapy and Gene Editing for β-Thalassemia by Christakopoulos, Georgios E., Telange, Rahul, Yen, Jonathan, Weiss, Mitchell J.

“…After many years of intensive research, emerging data from clinical trials indicate that gene therapy for transfusion-dependent β-thalassemia is now possible…”
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