未登录 A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels 一种靶向人PCSK9的强效表观遗传编辑器，用于持久降低低密度脂蛋白胆固醇水平 Tremblay, Frederic; Xiong, Qiang; Shah, Shrijal S.; Ko, Chih-Wei; Kelly, Kenneth; Morrison, Mary S.; Giancarlo, Cristiana; Ramirez, Ricardo N.; Hildebrand, Erica M.; Voytek, Sarah B.; El Sebae, Gabriel K.; Wright, Shane H.; Lofgren, Liam; Clarkson, Scott; Waters, Christine; Linder, Samantha J.; Liu, Songlei; Eom, Taesun; Parikh, Shefal; Weber, Yuki; Martinez, Salette; Malyala, Padma; Abubucker, Sahar; Friedland, Ari E.; Maeder, Morgan L.; Lombardo, Angelo; Myer, Vic E.; Jaffe, Aron B. 分享 收藏
An epigenetic editor targeting human PCSK9 efficiently and durably lowers Low Density Lipoprotein Cholesterol (LDL-C) in non-human primates Tremblay, F.; Kelly, K.; Shah, S.; El Sebae, G.; Ko, C. W.; Clarkson, S.; Ramirez, R. N.; Hildebrand, E.; Wright, S.; Morrison, M.; Voytek, S.; Abubucker, S.; Friedland, A.; Maeder, M.; Xiong, J. 分享 收藏
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10 Maeder, Morgan L.; Stefanidakis, Michael; Wilson, Christopher J.; Baral, Reshica; Barrera, Luis Alberto; Bounoutas, George S.; Bumcrot, David; Chao, Hoson; Ciulla, Dawn M.; DaSilva, Jennifer A.; Dass, Abhishek; Dhanapal, Vidya; Fennell, Tim J.; Friedland, Ari E.; Giannoukos, Georgia; Gloskowski, Sebastian W.; Glucksmann, Alexandra; Gotta, Gregory M.; Jayaram, Hariharan; Haskett, Scott J.; Hopkins, Bei; Horng, Joy E.; Joshi, Shivangi; Marco, Eugenio; Mepani, Rina; Reyon, Deepak; Ta, Terence; Tabbaa, Diana G.; Samuelsson, Steven J.; Shen, Shen; Skor, Maxwell N.; Stetkiewicz, Pam; Wang, Tongyao; Yudkoff, Clifford; Myer, Vic E.; Albright, Charles F.; Jiang, Haiyan 分享 收藏
Unexpected mutations after CRISPR-Cas9 editing in vivo Wilson, Christopher J.; Fennell, Tim; Bothmer, Anne; Maeder, Morgan L.; Reyon, Deepak; Cotta-Ramusino, Cecilia; Fernandez, Cecilia A.; Marco, Eugenio; Barrera, Luis A.; Jayaram, Hariharan; Albright, Charles F.; Cox, Gerald F.; Church, George M.; Myer, Vic E. 分享 收藏
UDiTaS™, a genome editing detection method for indels and genome rearrangements Giannoukos, Georgia; Ciulla, Dawn M.; Marco, Eugenio; Abdulkerim, Hayat S.; Barrera, Luis A.; Bothmer, Anne; Dhanapal, Vidya; Gloskowski, Sebastian W.; Jayaram, Hariharan; Maeder, Morgan L.; Skor, Maxwell N.; Wang, Tongyao; Myer, Vic E.; Wilson, Christopher J. 分享 收藏
Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Gene Editing Maeder, Morgan L.; Mepani, Rina; Gloskowski, Sebastian; Skor, Maxwell; Collins, McKensie; Shen, Shen; Gotta, Gregory; Marco, Eugenio; Barrera, Luis; Jayaram, Hari; Bumcrot, David 分享 收藏
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Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications Friedland, Ari E.; Baral, Reshica; Singhal, Pankhuri; Loveluck, Katherine; Shen, Shen; Sanchez, Minerva; Marco, Eugenio; Gotta, Gregory M.; Maeder, Morgan L.; Kennedy, Edward M.; Kornepati, Anand V. R.; Sousa, Alexander; Collins, McKensie A.; Jayaram, Hari; Cullen, Bryan R.; Bumcrot, David 分享 收藏
Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model Rahman, Shamim H.; Kuehle, Johannes; Reimann, Christian; Mlambo, Tafadzwa; Alzubi, Jamal; Maeder, Morgan L.; Riedel, Heimo; Fisch, Paul; Cantz, Tobias; Rudolph, Cornelia; Mussolino, Claudio; Joung, J. Keith; Schambach, Axel; Cathomen, Toni 分享 收藏
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Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing Maeder, Morgan L.; Shen, Shen; Burnight, Erin R.; Gloskowski, Sebastian; Mepani, Rina; Friedland, Ari E.; Jayaram, Hari; Gotta, Greg; Tucker, Budd A.; Bumcrot, David 分享 收藏
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo 阳离子脂质介导的蛋白质递送能够在体外和体内进行有效的基于蛋白质的基因组编辑 Zuris, John A.; Thompson, David B.; Shu, Yilai; Guilinger, John P.; Bessen, Jeffrey L.; Hu, Johnny H.; Maeder, Morgan L.; Joung, J. Keith; Chen, Zheng-Yi; Liu, David R. 分享 收藏
Pathways Disrupted in Human ALS Motor Neurons Identified through Genetic Correction of Mutant SOD1 (vol 14, pg 781, 2014) Kiskinis, Evangelos; Sandoe, Jackson; Williams, Luis A.; Boulting, Gabriella L.; Moccia, Rob; Wainger, Brian J.; Han, Steve; Peng, Theodore; Thams, Sebastian; Mikkilineni, Shravani; Mellin, Cassidy; Merkle, Florian T.; Davis-Dusenbery, Brandi N.; Ziller, Michael; Oakley, Derek; Ichida, Justin; Di Costanzo, Stefania; Atwater, Nick; Maeder, Morgan L.; Goodwin, Mathew J.; Nemesh, James; Handsaker, Robert E.; Paull, Daniel; Noggle, Scott; McCarroll, Steven A.; Joung, J. Keith; Woolf, Clifford J.; Brown, Robert H.; Eggan, Kevin 分享 收藏
Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins 使用可编程TALE-TET1融合蛋白靶向DNA去甲基化和激活内源基因 Maeder, Morgan L.; Angstman, James F.; Richardson, Marcy E.; Linder, Samantha J.; Cascio, Vincent M.; Tsai, Shengdar Q.; Ho, Quan H.; Sander, Jeffry D.; Reyon, Deepak; Bernstein, Bradley E.; Costello, Joseph F.; Wilkinson, Miles F.; Joung, J. Keith 分享 收藏
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TALEN-based Gene Correction for Epidermolysis Bullosa 基于TALEN的大疱性表皮松解症基因校正 Osborn, Mark J.; Starker, Colby G.; McElroy, Amber N.; Webber, Beau R.; Riddle, Megan J.; Xia, Lily; DeFeo, Anthony P.; Gabriel, Richard; Schmidt, Manfred; von Kalle, Christof; Carlson, Daniel F.; Maeder, Morgan L.; Joung, J. Keith; Wagner, John E.; Voytas, Daniel F.; Blazar, Bruce R.; Tolar, Jakub 分享 收藏
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