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Persistent repression of tau in the brain using engineered zinc finger protein transcription factors Wegmann, Susanne; DeVos, Sarah L.; Zeitler, Bryan; Marlen, Kimberly; Bennett, Rachel E.; Perez-Rando, Marta; MacKenzie, Danny; Yu, Qi; Commins, Caitlin; Bannon, Riley N.; Corjuc, Bianca T.; Chase, Alison; Diez, Lisa; Nguyen, Hoang-Oanh B.; Hinkley, Sarah; Zhang, Lei; Goodwin, Alicia; Ledeboer, Annemarie; Lam, Stephen; Ankoudinova, Irina; Hung Tran; Scarlott, Nicholas; Amora, Rainier; Surosky, Richard; Miller, Jeffrey C.; Robbins, Ashley B.; Rebar, Edward J.; Urnov, Fyodor D.; Holmes, Michael C.; Pooler, Amy M.; Riley, Brigit; Zhang, H. Steve; Hyman, Bradley T. 分享 收藏
Enhancing gene editing specificity by attenuating DNA cleavage kinetics Miller, Jeffrey C.; Patil, Deepak P.; Xia, Danny F.; Paine, Charles B.; Fauser, Friedrich; Richards, Hunter W.; Shivak, David A.; Bendana, Yuri R.; Hinkley, Sarah J.; Scarlott, Nicholas A.; Lam, Stephen C.; Reik, Andreas; Zhou, Yuanyue; Paschon, David E.; Li, Patrick; Wangzor, Tenzin; Lee, Gary; Zhang, Lei; Rebar, Edward J. 分享 收藏
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease Zeitler, Bryan; Froelich, Steven; Marlen, Kimberly; Shivak, David A.; Yu, Qi; Li, Davis; Pearl, Jocelynn R.; Miller, Jeffrey C.; Zhang, Lei; Paschon, David E.; Hinkley, Sarah J.; Ankoudinova, Irina; Lam, Stephen; Guschin, Dmitry; Kopan, Lexi; Cherone, Jennifer M.; Nguyen, Hoang-Oanh B.; Qiao, Guijuan; Ataei, Yasaman; Mendel, Matthew C.; Amora, Rainier; Surosky, Richard; Laganiere, Josee; Vu, B. Joseph; Narayanan, Anand; Sedaghat, Yalda; Tillack, Karsten; Thiede, Christina; Gaertner, Annette; Kwak, Seung; Bard, Jonathan; Mrzljak, Ladislav; Park, Larry; Heikkinen, Taneli; Lehtimaki, Kimmo K.; Svedberg, Marie M.; Haggkvist, Jenny; Tari, Lenke; Toth, Miklos; Varrone, Andrea; Halldin, Christer; Kudwa, Andrea E.; Ramboz, Sylvie; Day, Michelle; Kondapalli, Jyothisri; Surmeier, D. James; Urnov, Fyodor D.; Gregory, Philip D.; Rebar, Edward J.; Munoz-Sanjuan, Ignacio; Zhang, H. Steve 分享 收藏
Diversifying the structure of zinc finger nucleases for high-precision genome editing Paschon, David E.; Lussier, Stephanie; Wangzor, Tenzin; Xia, Danny F.; Li, Patrick W.; Hinkley, Sarah J.; Scarlott, Nicholas A.; Lam, Stephen C.; Waite, Adam J.; Truong, Lynn N.; Gandhi, Nimisha; Kadam, Bhakti N.; Patil, Deepak P.; Shivak, David A.; Lee, Gary K.; Holmes, Michael C.; Zhang, Lei; Miller, Jeffrey C.; Rebar, Edward J. 分享 收藏
Gene editing of the multi-copy H2A.B gene and its importance for fertility Anuar, Nur Diana; Kurscheid, Sebastian; Field, Matt; Zhang, Lei; Rebar, Edward; Gregory, Philip; Buchou, Thierry; Bowles, Josephine; Koopman, Peter; Tremethick, David J.; Soboleva, Tatiana A. 分享 收藏
Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo 线粒体中的基因组编辑在体内纠正了致病性mtDNA突变 Gammage, Payam A.; Viscomi, Carlo; Simard, Marie-Lune; Costa, Ana S. H.; Gaude, Edoardo; Powell, Christopher A.; Van Haute, Lindsey; McCann, Beverlyj; Rebelo-Guiomar, Pedro; Cerutti, Raffaele; Zhang, Lei; Rebar, Edward J.; Zeviani, Massimo; Frezza, Christian; Stewart, James B.; Minczuk, Michal 分享 收藏
DRUG DISCOVERY APPROACH FOR RARE NEUROLOGICAL DISEASES: USING NOVEL ZINC FINGER PROTEIN TECHNOLOGY TO DEVELOP POTENTIAL THERAPY FOR HUNTINGTON'S DISEASE Chiocco, Matthew; Choi, Vivian; Graham, Anne-Renee; Klatte, Debra; Francone, Omar; Thomson, David; Zeitler, Bryan; Froelich, Steven; Yu, Qi; Paschon, David E.; Miller, Jeffrey C.; Marlen, Kimberly; Rebar, Edward J.; Urnov, Fyodor D.; Gregory, Philip D.; Zhang, H. Steve 分享 收藏
Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects Laskowski, Tamara J.; Van Caeneghem, Yasmine; Pourebrahim, Rasoul; Ma, Chao; Ni, Zhenya; Garate, Zita; Crane, Ana M.; Li, Xuan Shirley; Liao, Wei; Gonzalez-Garay, Manuel; Carlos Segovia, Jose; Paschon, David E.; Rebar, Edward J.; Holmes, Michael C.; Kaufman, Dan; Vandekerckhove, Bart; Davis, Brian R. 分享 收藏
Highly Efficient, ZFN-Driven Knockout of Surface Expression of the T-Cell Receptor and HLA Class I Proteins in Human T-Cells for Enhancing Allogeneic Adoptive Cell Therapies Lee, Gary K.; Liu, Pei-qi; Gandhi, Nimisha; Truong, Lynn; Reik, Andreas; Lam, Stephen; Hinkley, Sarah J.; Miller, Jeffrey C.; Paschon, David E.; Zhang, Lei; Rebar, Edward J.; Holmes, Michael C. 分享 收藏
Genetic editing of HLA expression in hematopoietic stem cells to broaden their human application Torikai, Hiroki; Mi, Tiejuan; Gragert, Loren; Maiers, Martin; Najjar, Amer; Ang, Sonny; Maiti, Sourindra; Dai, Jianliang; Switzer, Kirsten C.; Huls, Helen; Dulay, Gladys P.; Reik, Andreas; Rebar, Edward J.; Holmes, Michael C.; Gregory, Philip D.; Champlin, Richard E.; Shpall, Elizabeth J.; Cooper, Laurence J. N. 分享 收藏
Mammalian interspecies substitution of immune modulatory alleles by genome editing Lillico, Simon G.; Proudfoot, Chris; King, Tim J.; Tan, Wenfang; Zhang, Lei; Mardjuki, Rachel; Paschon, David E.; Rebar, Edward J.; Urnov, Fyodor D.; Mileham, Alan J.; McLaren, David G.; Whitelaw, C. Bruce A. 分享 收藏
Multi-reporter selection for the design of active and more specific zinc-finger nucleases for genome editing Oakes, Benjamin L.; Xia, Danny F.; Rowland, Elizabeth F.; Xu, Denise J.; Ankoudinova, Irina; Borchardt, Jennifer S.; Zhang, Lei; Li, Patrick; Miller, Jeffrey C.; Rebar, Edward J.; Noyes, Marcus B. 分享 收藏
Functional footprinting of regulatory DNA Vierstra, Jeff; Reik, Andreas; Chang, Kai-Hsin; Stehling-Sun, Sandra; Zhou, Yuanyue; Hinkley, Sarah J.; Paschon, David E.; Zhang, Lei; Psatha, Nikoletta; Bendana, Yuri R.; O'Neil, Colleen M.; Song, Alexander H.; Mich, Andrea K.; Liu, Pei-Qi; Lee, Gary; Bauer, Daniel E.; Holmes, Michael C.; Orkin, Stuart H.; Papayannopoulo, Thalia; Stamatoyannopoulos, George; Rebar, Edward J.; Gregory, Philip D.; Urnov, Fyodor D.; Stamatoyannopoulos, John A. 分享 收藏
Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma Beane, Joal D.; Lee, Gary; Zheng, Zhili; Mendel, Matthew; Abate-Daga, Daniel; Bharathan, Mini; Black, Mary; Gandhi, Nimisha; Yu, Zhiya; Chandran, Smita; Giedlin, Martin; Ando, Dale; Miller, Jeff; Paschon, David; Guschin, Dmitry; Rebar, Edward J.; Reik, Andreas; Holmes, Michael C.; Gregory, Philip D.; Restifo, Nicholas P.; Rosenberg, Steven A.; Morgan, Richard A.; Feldman, Steven A. 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
Preclinical Studies for the First Hematopoietic Stem Cell (HSC) Gene Editing Trial: Phase 1 Study of Beta-Thalassemia With Autologous Transplantation of Zinc Finger Nuclease-Treated HSC To Upregulate Fetal Hemoglobin Ando, Dale; Walters, Mark C.; Chang, Kai-Hsin; Reik, Andreas; Urnov, Fyodor D.; Lee, Gary; Spratt, Kaye S.; Rebar, Edward J.; Gregory, Philip D.; Tang, Winson; Papayannopoulou, Thalia; Stamatoyannopoulos, George; Nichol, Geoffrey 分享 收藏
From GWAS To the Clinic: Genome-Editing the Human BCL11A Erythroid Enhancer for Fetal Globin Elevation in the Hemoglobinopathies Reik, Andreas; Chang, Kai-Hsin; Vierstra, Jeff; Psatha, Nikoletta; Stehling-Sun, Sandra; Zhou, Yuanyue; O'Neil, Colleen M.; Mich, Andrea; Miller, Jeffrey C.; Lee, Gary; Tan, Siyuan; Jiang, Haiyan; Stamatoyannopoulos, George; Papayannopoulou, Thalia; Rebar, Edward J.; Gregory, Philip D.; Stamatoyannopoulos, John A.; Urnov, Fyodor D. 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
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