Use of zinc-finger nucleases to knock out the WAS gene in K562 cells: a human cellular model for Wiskott-Aldrich syndrome by Miguel G. Toscano, Per Anderson, Pilar Muñoz, Gema Lucena, Marién Cobo, Karim Benabdellah, Philip D. Gregory, Michael C. Holmes, Francisco Martin

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Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy. by Christopher W Peterson, Jianbin Wang, Claire Deleage, Sowmya Reddy, Jasbir Kaur, Patricia Polacino, Andreas Reik, Meei-Li Huang, Keith R Jerome, Shiu-Lok Hu, Michael C Holmes, Jacob D Estes, Hans-Peter Kiem

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DNA ligase III promotes alternative nonhomologous end-joining during chromosomal translocation formation. by Deniz Simsek, Erika Brunet, Sunnie Yan-Wai Wong, Sachin Katyal, Yankun Gao, Peter J McKinnon, Jacqueline Lou, Lei Zhang, James Li, Edward J Rebar, Philip D Gregory, Michael C Holmes, Maria Jasin

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Efficient genome editing in hematopoietic stem cells with helper-dependent Ad5/35 vectors expressing site-specific endonucleases under microRNA regulation by Kamola Saydaminova, Xun Ye, Hongjie Wang, Maximilian Richter, Martin Ho, HongZhuan Chen, Ning Xu, Jin-Soo Kim, Eirini Papapetrou, Michael C Holmes, Philip D Gregory, Donna Palmer, Philip Ng, Anja Ehrhardt, André Lieber

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AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction by Makiko Yasuda, Marshall W. Huston, Silvere Pagant, Lin Gan, Susan St. Martin, Scott Sproul, Daniel Richards, Stephen Ballaron, Khaled Hettini, Annemarie Ledeboer, Lillian Falese, Liching Cao, Yanmei Lu, Michael C. Holmes, Kathleen Meyer, Robert J. Desnick, Thomas Wechsler

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Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects by Tamara J. Laskowski, Yasmine Van Caeneghem, Rasoul Pourebrahim, Chao Ma, Zhenya Ni, Zita Garate, Ana M. Crane, Xuan Shirley Li, Wei Liao, Manuel Gonzalez-Garay, Jose Carlos Segovia, David E. Paschon, Edward J. Rebar, Michael C. Holmes, Dan Kaufman, Bart Vandekerckhove, Brian R. Davis

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Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells by David L DiGiusto, Paula M Cannon, Michael C Holmes, Lijing Li, Anitha Rao, Jianbin Wang, Gary Lee, Philip D. Gregory, Kenneth A Kim, Samuel B Hayward, Kathleen Meyer, Colin Exline, Evan Lopez, Jill Henley, Nancy Gonzalez, Victoria Bedell, Rodica Stan, John A Zaia

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Supraphysiologic control over HIV-1 replication mediated by CD8 T cells expressing a re-engineered CD4-based chimeric antigen receptor. by Rachel S Leibman, Max W Richardson, Christoph T Ellebrecht, Colby R Maldini, Joshua A Glover, Anthony J Secreto, Irina Kulikovskaya, Simon F Lacey, Sarah R Akkina, Yanjie Yi, Farida Shaheen, Jianbin Wang, Keith A Dufendach, Michael C Holmes, Ronald G Collman, Aimee S Payne, James L Riley

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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells by Kai-Hsin Chang, Sarah E. Smith, Timothy Sullivan, Kai Chen, Qianhe Zhou, Jason A. West, Mei Liu, Yingchun Liu, Benjamin F. Vieira, Chao Sun, Vu P. Hong, Mingxuan Zhang, Xiao Yang, Andreas Reik, Fyodor D. Urnov, Edward J. Rebar, Michael C. Holmes, Olivier Danos, Haiyan Jiang, Siyuan Tan

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Targeted gene therapy and cell reprogramming in Fanconi anemia by Paula Rio, Rocio Baños, Angelo Lombardo, Oscar Quintana‐Bustamante, Lara Alvarez, Zita Garate, Pietro Genovese, Elena Almarza, Antonio Valeri, Begoña Díez, Susana Navarro, Yaima Torres, Juan P Trujillo, Rodolfo Murillas, Jose C Segovia, Enrique Samper, Jordi Surralles, Philip D Gregory, Michael C Holmes, Luigi Naldini, Juan A Bueren

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Potent and Broad Inhibition of HIV-1 by a Peptide from the gp41 Heptad Repeat-2 Domain Conjugated to the CXCR4 Amino Terminus. by George J Leslie, Jianbin Wang, Max W Richardson, Beth S Haggarty, Kevin L Hua, Jennifer Duong, Anthony J Secreto, Andrea P O Jordon, Josephine Romano, Kritika E Kumar, Joshua J DeClercq, Philip D Gregory, Carl H June, Michael J Root, James L Riley, Michael C Holmes, James A Hoxie

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Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells by Ana M. Crane, Philipp Kramer, Jacquelin H. Bui, Wook Joon Chung, Xuan Shirley Li, Manuel L. Gonzalez-Garay, Finn Hawkins, Wei Liao, Daniela Mora, Sangbum Choi, Jianbin Wang, Helena C. Sun, David E. Paschon, Dmitry Y. Guschin, Philip D. Gregory, Darrell N. Kotton, Michael C. Holmes, Eric J. Sorscher, Brian R. Davis

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Diversifying the structure of zinc finger nucleases for high-precision genome editing by David E. Paschon, Stephanie Lussier, Tenzin Wangzor, Danny F. Xia, Patrick W. Li, Sarah J. Hinkley, Nicholas A. Scarlott, Stephen C. Lam, Adam J. Waite, Lynn N. Truong, Nimisha Gandhi, Bhakti N. Kadam, Deepak P. Patil, David A. Shivak, Gary K. Lee, Michael C. Holmes, Lei Zhang, Jeffrey C. Miller, Edward J. Rebar

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Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases. by Craig B Wilen, Jianbin Wang, John C Tilton, Jeffrey C Miller, Kenneth A Kim, Edward J Rebar, Scott A Sherrill-Mix, Sean C Patro, Anthony J Secreto, Andrea P O Jordan, Gary Lee, Joshua Kahn, Pyone P Aye, Bruce A Bunnell, Andrew A Lackner, James A Hoxie, Gwenn A Danet-Desnoyers, Frederic D Bushman, James L Riley, Philip D Gregory, Carl H June, Michael C Holmes, Robert W Doms

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