LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson's disease patients: Reversal by gene correction
Sanders, Laurie H, Laganière, Josée, Cooper, Oliver, Mak, Sally K, Vu, B. Joseph, Huang, Y. Anne, Paschon, David E, Vangipuram, Malini, Sundararajan, Ramya, Urnov, Fyodor D, Langston, J. William, Gregory, Philip D, Zhang, H. Steve, Greenamyre, J. Timothy, Isacson, Ole, Schüle, Birgitt
Published in Neurobiology of disease (01.02.2014)
Published in Neurobiology of disease (01.02.2014)
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In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Sharma, Rajiv, Anguela, Xavier M., Doyon, Yannick, Wechsler, Thomas, DeKelver, Russell C., Sproul, Scott, Paschon, David E., Miller, Jeffrey C., Davidson, Robert J., Shivak, David, Zhou, Shangzhen, Rieders, Julianne, Gregory, Philip D., Holmes, Michael C., Rebar, Edward J., High, Katherine A.
Published in Blood (08.10.2015)
Published in Blood (08.10.2015)
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Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells
Yusa, Kosuke, Rashid, S. Tamir, Strick-Marchand, Helene, Varela, Ignacio, Liu, Pei-Qi, Paschon, David E., Miranda, Elena, Ordóñez, Adriana, Hannan, Nicholas R. F., Rouhani, Foad J., Darche, Sylvie, Alexander, Graeme, Marciniak, Stefan J., Fusaki, Noemi, Hasegawa, Mamoru, Holmes, Michael C., Di Santo, James P., Lomas, David A., Bradley, Allan, Vallier, Ludovic
Published in Nature (London) (20.10.2011)
Published in Nature (London) (20.10.2011)
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Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
Hoban, Megan D., Cost, Gregory J., 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.
Published in Blood (23.04.2015)
Published in Blood (23.04.2015)
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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.
Published in Nature communications (08.03.2019)
Published in Nature communications (08.03.2019)
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Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
Conway, Anthony, Mendel, Matthew, Kim, Kenneth, McGovern, Kyle, Boyko, Alisa, Zhang, Lei, Miller, Jeffrey C., DeKelver, Russell C., Paschon, David E., Mui, Barbara L., Lin, Paulo J.C., Tam, Ying K., Barbosa, Chris, Redelmeier, Tom, Holmes, Michael C., Lee, Gary
Published in Molecular therapy (10.04.2019)
Published in Molecular therapy (10.04.2019)
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Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer
Provasi, Elena, Genovese, Pietro, Lombardo, Angelo, Magnani, Zulma, Liu, Pei-Qi, Reik, Andreas, Chu, Victoria, Paschon, David E, Zhang, Lei, Kuball, Jurgen, Camisa, Barbara, Bondanza, Attilio, Casorati, Giulia, Ponzoni, Maurilio, Ciceri, Fabio, Bordignon, Claudio, Greenberg, Philip D, Holmes, Michael C, Gregory, Philip D, Naldini, Luigi, Bonini, Chiara
Published in Nature medicine (01.05.2012)
Published in Nature medicine (01.05.2012)
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A TALE nuclease architecture for efficient genome editing
Rebar, Edward J, Miller, Jeffrey C, Tan, Siyuan, Qiao, Guijuan, Barlow, Kyle A, Wang, Jianbin, Xia, Danny F, Meng, Xiangdong, Paschon, David E, Leung, Elo, Hinkley, Sarah J, Dulay, Gladys P, Hua, Kevin L, Ankoudinova, Irina, Cost, Gregory J, Urnov, Fyodor D, Zhang, H Steve, Holmes, Michael C, Zhang, Lei, Gregory, Philip D
Published in Nature biotechnology (01.02.2011)
Published in Nature biotechnology (01.02.2011)
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Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells
Crane, Ana M, Kramer, Philipp, Bui, Jacquelin H, Chung, Wook Joon, Li, Xuan Shirley, Gonzalez-Garay, Manuel L, Hawkins, Finn, Liao, Wei, Mora, Daniela, Choi, Sangbum, Wang, Jianbin, Sun, Helena C, Paschon, David E, Guschin, Dmitry Y, Gregory, Philip D, Kotton, Darrell N, Holmes, Michael C, Sorscher, Eric J, Davis, Brian R
Published in Stem cell reports (14.04.2015)
Published in Stem cell reports (14.04.2015)
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Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease
De Ravin, Suk See, Reik, Andreas, Liu, Pei-Qi, Li, Linhong, Wu, Xiaolin, Su, Ling, Raley, Castle, Theobald, Narda, Choi, Uimook, Song, Alexander H, Chan, Andy, Pearl, Jocelynn R, Paschon, David E, Lee, Janet, Newcombe, Hannah, Koontz, Sherry, Sweeney, Colin, Shivak, David A, Zarember, Kol A, Peshwa, Madhusudan V, Gregory, Philip D, Urnov, Fyodor D, Malech, Harry L
Published in Nature biotechnology (01.04.2016)
Published in Nature biotechnology (01.04.2016)
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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, Gärtner, Annette, Kwak, Seung, Bard, Jonathan, Mrzljak, Ladislav, Park, Larry, Heikkinen, Taneli, Lehtimäki, Kimmo K, Svedberg, Marie M, Häggkvist, Jenny, Tari, Lenke, Tóth, Miklós, 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, Muñoz-Sanjuán, Ignacio, Zhang, H Steve
Published in Nature medicine (01.07.2019)
Published in Nature medicine (01.07.2019)
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In vivo genome editing restores haemostasis in a mouse model of haemophilia
Li, Hojun, Haurigot, Virginia, Doyon, Yannick, Li, Tianjian, Wong, Sunnie Y., Bhagwat, Anand S., Malani, Nirav, Anguela, Xavier M., Sharma, Rajiv, Ivanciu, Lacramiora, Murphy, Samuel L., Finn, Jonathan D., Khazi, Fayaz R., Zhou, Shangzhen, Paschon, David E., Rebar, Edward J., Bushman, Frederic D., Gregory, Philip D., Holmes, Michael C., High, Katherine A.
Published in Nature (London) (14.07.2011)
Published in Nature (London) (14.07.2011)
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Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells
Drubin, David G, Lee, Andrew H, Cheng, Aaron T, Doyon, Yannick, Vo, Thuy D, Miller, Jeffrey C, Cherone, Jennifer M, Cheng, Jackie, Santiago, Yolanda, Urnov, Fyodor D, Paschon, David E, Doyon, Jeffrey B, Zhang, Lei, Rebar, Edward J, Gregory, Philip D, Zeitler, Bryan
Published in Nature cell biology (01.03.2011)
Published in Nature cell biology (01.03.2011)
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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.
Published in Scientific reports (22.02.2016)
Published in Scientific reports (22.02.2016)
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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, Papayannopoulou, Thalia, Stamatoyannopoulos, George, Rebar, Edward J, Gregory, Philip D, Urnov, Fyodor D, Stamatoyannopoulos, John A
Published in Nature methods (01.10.2015)
Published in Nature methods (01.10.2015)
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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., Bendaña, 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.
Published in Nature biotechnology (01.08.2019)
Published in Nature biotechnology (01.08.2019)
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Improved specificity of TALE-based genome editing using an expanded RVD repertoire
Miller, Jeffrey C, Zhang, Lei, Xia, Danny F, Campo, John J, Ankoudinova, Irina V, Guschin, Dmitry Y, Babiarz, Joshua E, Meng, Xiangdong, Hinkley, Sarah J, Lam, Stephen C, Paschon, David E, Vincent, Anna I, Dulay, Gladys P, Barlow, Kyle A, Shivak, David A, Leung, Elo, Kim, Jinwon D, Amora, Rainier, Urnov, Fyodor D, Gregory, Philip D, Rebar, Edward J
Published in Nature methods (01.05.2015)
Published in Nature methods (01.05.2015)
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Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome
DeKelver, Russell C, Choi, Vivian M, Moehle, Erica A, Paschon, David E, Hockemeyer, Dirk, Meijsing, Sebastiaan H, Sancak, Yasemin, Cui, Xiaoxia, Steine, Eveline J, Miller, Jeffrey C, Tam, Phillip, Bartsevich, Victor V, Meng, Xiangdong, Rupniewski, Igor, Gopalan, Sunita M, Sun, Helena C, Pitz, Kathleen J, Rock, Jeremy M, Zhang, Lei, Davis, Gregory D, Rebar, Edward J, Cheeseman, Iain M, Yamamoto, Keith R, Sabatini, David M, Jaenisch, Rudolf, Gregory, Philip D, Urnov, Fyodor D
Published in Genome research (01.08.2010)
Published in Genome research (01.08.2010)
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Efficient targeted gene disruption in the soma and germ line of the frog Xenopus tropicalis using engineered zinc-finger nucleases
Young, John J, Cherone, Jennifer M, Doyon, Yannick, Ankoudinova, Irina, Faraji, Farhoud M, Lee, Andrew H, Ngo, Catherine, Guschin, Dmitry Y, Paschon, David E, Miller, Jeffrey C, Zhang, Lei, Rebar, Edward J, Gregory, Philip D, Urnov, Fyodor D, Harland, Richard M, Zeitler, Bryan
Published in Proceedings of the National Academy of Sciences - PNAS (26.04.2011)
Published in Proceedings of the National Academy of Sciences - PNAS (26.04.2011)
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Humanized Mouse Model of HIV-1 Latency with Enrichment of Latent Virus in PD-1 + and TIGIT + CD4 T Cells
Llewellyn, George N, Seclén, Eduardo, Wietgrefe, Stephen, Liu, Siyu, Chateau, Morgan, Pei, Hua, Perkey, Katherine, Marsden, Matthew D, Hinkley, Sarah J, Paschon, David E, Holmes, Michael C, Zack, Jerome A, Louie, Stan G, Haase, Ashley T, Cannon, Paula M
Published in Journal of virology (15.05.2019)
Published in Journal of virology (15.05.2019)
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