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Integration of therapeutic cargo into the human genome with programmable type V-K CAST 利用可编程V-K型CAST将治疗性载荷整合到人类基因组中 Liu, Jason; Goltsman, Daniela S. Aliaga; Alexander, Lisa M.; Khayi, Khak Khak; Hong, Jennifer H.; Dunham, Drew T.; Romano, Christine A.; Temoche-Diaz, Morayma M.; Chadha, Shailaja; Ocampo, Rodrigo Fregoso; Oki-O'Connell, Jennifer; Janson, Owen P.; Turcios, Keirstinne; Gonzalez-Osorio, Liliana; Muysson, Jared; Rahman, Jenat; Laperriere, Sarah M.; Devoto, Audra E.; Castelle, Cindy J.; Butterfield, Cristina N.; Cost, Gregory J.; Brown, Christopher T.; Thomas, Brian C. 分享 收藏
AFNT-212: A TRAC-knocked-in KRASG12D-specific TCR-T cell product enhanced with CD8αß and a chimeric cytokine receptor for treatment of solid cancers Drain, Allison; Rouillard, Nicholas; Swanson, Nathaniel; Canestraro, Martina; Narayan, Santosh; Warner, Tyler; Danek, Nicole; Gareau, Ken; Liang, Jinsheng; Shen, Luhua; Tetrault, Tanya; Priyata, Iqraa; Vidyasagar, Sarah; Riggins-Walker, Taylor; Liu, Hui-Wen; Pechhold, Klaus; Brown, Lauren; Francis, Joshua; He, Xingyue; Browne, Patrick; Lamothe, Rebecca; Storlie, Meghan; Cost, Gregory; Schmitt, Thomas M.; Greenberg, Philip D.; Chandran, Smita S.; Klebanoff, Christopher A.; Lam, Hubert; Gupta, Ankit; Hallet, Damien; Shapiro, Gary; Nguyen, Kim; Vincent, Loic 分享 收藏
Non-viral engineered T cell therapy specific for the hotspot mutation p53 R175H that integrates signal 1 (TCR), signal 2 ( co-stimulation) and signal 3 (cytokine) and co-opts FasL-dependent apoptosis to achieve a coordinated antitumor CD4/8 T cell response Narayan, Santosh; Gareau, Ken; Gupta, Ankit; Ferrell, Josh; Rouillard, Nicholas; Warner, Tyler; Liang, Jinsheng; Shen, Luhua; Tetrault, Tanya; Francis, Joshua; He, Xingyue; Browne, Patrick J.; Lamothe, Rebecca; Storlie, Meghan D.; Cost, Gregory J.; Schmitt, Thomas M.; Greenberg, Philip D.; Chandran, Smita S.; Hallet, Damien; Gormally, Michael; Klebanoff, Chistopher A.; Shapiro, Gary; Nguyen, Kim; Vincent, Loic 分享 收藏
Novel and Engineered Type II CRISPR Systems from Uncultivated Microbes with Broad Genome Editing Capability Alexander, Lisa M. M.; Goltsman, Daniela S. Aliaga S.; Liu, Jason; Lin, Jyun-Liang; Temoche-Diaz, Morayma M. M.; Laperriere, Sarah M. M.; Neerincx, Andreas; Bednarski, Christien; Knyphausen, Philipp; Cohnen, Andre; Albers, Justine; Gonzalez-Osorio, Liliana; Ocampo, Rodrigo Fregoso; Oki, Jennifer; Devoto, Audra E. E.; Castelle, Cindy J. J.; Lamothe, Rebecca C. C.; Cost, Gregory J. J.; Butterfield, Cristina N. N.; Thomas, Brian C. C.; Brown, Christopher T. T. 分享 收藏
Novel CRISPR-Associated Gene-Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genome Engineering Lamothe, Rebecca C.; Storlie, Meghan D.; Espinosa, Diego A.; Rudlaff, Rachel; Browne, Patrick; Liu, Jason; Rivas, Andres; Devoto, Audra; Oki, Jennifer; Khoubyari, Ashcon; Goltsman, Daniela S. Aliaga; Lin, Jyun-Liang; Butterfield, Cristina N.; Brown, Christopher T.; Thomas, Brian C.; Cost, Gregory J. 分享 收藏
Resolution of hepatic fibrosis after ZFN-mediated gene editing in the PiZ mouse model of human α1-antitrypsin deficiency Li, Yanfeng; Guha, Chandan; Asp, Patrik; Wang, Xia; Tchaikovskya, Tatyana L.; Kim, Kenneth; Mendel, Matthew; Cost, Gregory J.; Perlmutter, David H.; Roy-Chowdhury, Namita; Fox, Ira J.; Conway, Anthony; Roy-Chowdhury, Jayanta 分享 收藏
Compact Cas9d and HEARO enzymes for genome editing discovered from uncultivated microbes Goltsman, Daniela S. Aliaga; Alexander, Lisa M.; Lin, Jyun-Liang; Fregoso Ocampo, Rodrigo; Freeman, Benjamin; Lamothe, Rebecca C.; Perez Rivas, Andres; Temoche-Diaz, Morayma M.; Chadha, Shailaja; Nordenfelt, Natalie; Janson, Owen P.; Barr, Ian; Devoto, Audra E.; Cost, Gregory J.; Butterfield, Cristina N.; Thomas, Brian C.; Brown, Christopher T. 分享 收藏
Successful knock-in of Hypertrophic Cardiomyopathy-mutation R723G into the MYH7 gene mimics HCM pathology in pigs Montag, J.; Petersen, B.; Floegel, A. K.; Becker, E.; Lucas-Hahn, A.; Cost, G. J.; Muehlfeld, C.; Kraft, T.; Niemann, H.; Brenner, B. 分享 收藏
A Genome Edited Pig with the Hypertrophic Cardiomyopathy-Mutation R723G in the MYH7-gene Montag, Judith; Petersen, Bjorn; Floegel, Anna Katharina; Becker, Edgar; Lucas-Hahn, Andrea; Cost, Gregory J.; Muehlfeld, Christian; Kraft, Theresia; Niemann, Heiner; Brenner, Bernhard 分享 收藏
Towards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 Mutations Genovese, Pietro; Schiroli, Giulia; Conway, Anthony; Ferrari, Samuele; Guschin, Dmitry Y.; Di Tomaso, Tiziano; Albano, Luisa; Marin, Virna; Giuliani, Erica; Gregory, Philip D.; Holmes, Michael C.; Gentner, Bernhard; Bovolenta, Chiara; Cost, Gregory J.; Lombardo, Angelo; Naldini, Luigi 分享 收藏
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A NOVEL IN VIVO CHEMOSELECTION STRATEGY FOR GENETICALLY MODIFIED HEMATOPOIETIC STEM CELLS Hacke, K.; Treger, J.; Bogan, B.; Rezek, V.; Narukawa, M.; Shimizu, S.; Liu, P-Q; Reik, A.; Lieber, A.; Crooks, G.; Kohn, D.; An, D. S.; Kitchen, S.; Gregory, P.; Cost, G.; Holmes, M.; Kasahara, N. 分享 收藏
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Correction of the Sickle-Cell Disease Mutation in Human Hematopoietic Stem/Progenitor Cells Hoban, Megan D.; Mendel, Matthew C.; Romero, Zulema; Kaufman, Michael L.; Joglekar, Alok V.; Ho, Michelle; Lumaquin, Dianne; Gray, David; Lill, Georgia R.; Cooper, Aaron R.; Urbinati, Fabrizia; Senadheera, Shantha; Zhu, Allen; Liu, Pei-Qi; Paschon, David E.; Zhang, Lei; Rebar, Edward J.; Wilber, Andrew; Wang, Xiaoyan; Gregory, Philip D.; Holmes, Michael C.; Reik, Andreas; Hollis, Roger P.; Kohn, Donald B.; Cost, Gregory J. 分享 收藏
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Genome Editing-Enabled HIS Assays Expand Drug Target Pathways for Charcot-Marie-Tooth Disease Inglese, James; Dranchak, Patricia; Moran, John J.; Jang, Sung-Wook; Srinivasan, Rajini; Santiago, Yolanda; Zhang, Lei; Guha, Rajarshi; Martinez, Natalia; MacArthur, Ryan; Cost, Gregory J.; Svaren, John 分享 收藏
Efficient gene targeting by homology-directed repair in rat zygotes using TALE nucleases Remy, Severine; Tesson, Laurent; Menoret, Severine; Usal, Claire; De Cian, Anne; Thepenier, Virginie; Thinard, Reynald; Baron, Daniel; Charpentier, Marine; Renaud, Jean-Baptiste; Buelow, Roland; Cost, Gregory J.; Giovannangeli, Carine; Fraichard, Alexandre; Concordet, Jean-Paul; Anegon, Ignacio 分享 收藏
Human Intestinal Tissue with Adult Stem Cell Properties Derived from Pluripotent Stem Cells (vol 2, pg 838, 2014) Forster, Ryan; Chiba, Kunitoshi; Schaeffer, Lorian; Regalado, Samuel G.; Lai, Christine S.; Gao, Qing; Kiani, Samira; Farin, Henner F.; Clevers, Hans; Cost, Gregory J.; Chan, Andy; Rebar, Edward J.; Urnov, Fyodor D.; Gregory, Philip D.; Pachter, Lior; Jaenisch, Rudolf; Hockemeyer, Dirk 分享 收藏
Human Intestinal Tissue with Adult Stem Cell Properties Derived from Pluripotent Stem Cells Forster, Ryan; Chiba, Kunitoshi; Schaeffer, Lorian; Regalado, Samuel G.; Lai, Christine S.; Gao, Qing; Kiani, Samira; Farin, Henner F.; Clevers, Hans; Cost, Gregory J.; Chan, Andy; Rebar, Edward J.; Urnov, Fyodor D.; Gregory, Philip D.; Pachter, Lior; Jaenisch, Rudolf; Hockemeyer, Dirk 分享 收藏
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