Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation

by Benabdallah, Basma F, Duval, Arnaud, Rousseau, Joel, Chapdelaine, Pierre, Holmes, Michael C, Haddad, Eli, Tremblay, Jacques P, Beauséjour, Christian M
Published 2013

Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo

by Holt, Nathalia, Wang, Jianbin, Kim, Kenneth, Friedman, Geoffrey, Wang, Xingchao, Taupin, Vanessa, Crooks, Gay M., Kohn, Donald B., Gregory, Philip D., Holmes, Michael C., Cannon, Paula M.
Published 2010

Use of zinc-finger nucleases to knock out the WAS gene in K562 cells: a human cellular model for Wiskott-Aldrich syndrome

by Toscano, Miguel G., Anderson, Per, Muñoz, Pilar, Lucena, Gema, Cobo, Marién, Benabdellah, Karim, Gregory, Philip D., Holmes, Michael C., Martin, Francisco
Published 2013

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery

by Wang, Jianbin, DeClercq, Joshua J., Hayward, Samuel B., Li, Patrick Wai-Lun, Shivak, David A., Gregory, Philip D., Lee, Gary, Holmes, Michael C.
Published 2016

DNA Ligase III Promotes Alternative Nonhomologous End-Joining during Chromosomal Translocation Formation

by Simsek, Deniz, Brunet, Erika, Wong, Sunnie Yan-Wai, Katyal, Sachin, Gao, Yankun, McKinnon, Peter J., Lou, Jacqueline, Zhang, Lei, Li, James, Rebar, Edward J., Gregory, Philip D., Holmes, Michael C., Jasin, Maria
Published 2011

Homology-driven genome editing in hematopoietic stem and progenitor cells using zinc finger nuclease mRNA and AAV6 donors

by Wang, Jianbin, Exline, Colin M., DeClercq, Joshua J., Llewellyn, G. Nicholas, Hayward, Samuel B., Li, Patrick Wai-Lun, Shivak, David A., Surosky, Richard T., Gregory, Philip D., Holmes, Michael C., Cannon, Paula M
Published 2015

Absence of WASp Enhances Hematopoietic and Megakaryocytic Differentiation in a Human Embryonic Stem Cell Model

by Toscano, Miguel G, Muñoz, Pilar, Sánchez-Gilabert, Almudena, Cobo, Marién, Benabdellah, Karim, Anderson, Per, Ramos-Mejía, Verónica, Real, Pedro J, Neth, Olaf, Molinos-Quintana, Agueda, Gregory, Philip D, Holmes, Michael C, Martin, Francisco
Published 2016

Efficient genome editing in hematopoietic stem cells with helper-dependent Ad5/35 vectors expressing site-specific endonucleases under microRNA regulation

by Saydaminova, Kamola, Ye, Xun, Wang, Hongjie, Richter, Maximilian, Ho, Martin, Chen, HongZhuan, Xu, Ning, Kim, Jin-Soo, Papapetrou, Eirini, Holmes, Michael C, Gregory, Philip D, Palmer, Donna, Ng, Philip, Ehrhardt, Anja, Lieber, André
Published 2015

Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells

by Genovese, Pietro, Schiroli, Giulia, Escobar, Giulia, Tomaso, Tiziano Di, Firrito, Claudia, Calabria, Andrea, Moi, Davide, Mazzieri, Roberta, Bonini, Chiara, Holmes, Michael C., Gregory, Philip D., van der Burg, Mirjam, Gentner, Bernhard, Montini, Eugenio, Lombardo, Angelo, Naldini, Luigi
Published 2014

In vivo genome editing restores hemostasis in a mouse model of hemophilia

by 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 2011

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

by Yusa, Kosuke, Rashid, S. Tamir, Strick-Marchand, Helene, Varela, Ignacio, Liu, Pei-Qi, Paschon, David E., Miranda, Elena, Ordóñez, Adriana, Hannan, Nick, Rouhani, Foad Jafari, 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 2011

Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger Nucleases

by Li, Lijing, Krymskaya, Ludmila, Wang, Jianbin, Henley, Jill, Rao, Anitha, Cao, Lan-Feng, Tran, Chy-Anh, Torres-Coronado, Monica, Gardner, Agnes, Gonzalez, Nancy, Kim, Kenneth, Liu, Pei-Qi, Hofer, Ursula, Lopez, Evan, Gregory, Philip D, Liu, Qing, Holmes, Michael C, Cannon, Paula M, Zaia, John A, DiGiusto, David L
Published 2013

Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells

by DiGiusto, David L, Cannon, Paula M, Holmes, Michael C, Li, Lijing, Rao, Anitha, Wang, Jianbin, Lee, Gary, Gregory, Philip D., Kim, Kenneth A, Hayward, Samuel B, Meyer, Kathleen, Exline, Colin, Lopez, Evan, Henley, Jill, Gonzalez, Nancy, Bedell, Victoria, Stan, Rodica, Zaia, John A
Published 2016

Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects

by 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, Segovia, Jose Carlos, Paschon, David E., Rebar, Edward J., Holmes, Michael C., Kaufman, Dan, Vandekerckhove, Bart, Davis, Brian R.
Published 2016

Potent and Broad Inhibition of HIV-1 by a Peptide from the gp41 Heptad Repeat-2 Domain Conjugated to the CXCR4 Amino Terminus

by Leslie, George J., Wang, Jianbin, Richardson, Max W., Haggarty, Beth S., Hua, Kevin L., Duong, Jennifer, Secreto, Anthony J., Jordon, Andrea P. O., Romano, Josephine, Kumar, Kritika E., DeClercq, Joshua J., Gregory, Philip D., June, Carl H., Root, Michael J., Riley, James L., Holmes, Michael C., Hoxie, James A.
Published 2016

Genetic editing of HLA expression in hematopoietic stem cells to broaden their human application

by 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.
Published 2016

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

by 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 2015

Targeted gene therapy and cell reprogramming in Fanconi anemia

by Rio, Paula, Baños, Rocio, Lombardo, Angelo, Quintana-Bustamante, Oscar, Alvarez, Lara, Garate, Zita, Genovese, Pietro, Almarza, Elena, Valeri, Antonio, Díez, Begoña, Navarro, Susana, Torres, Yaima, Trujillo, Juan P, Murillas, Rodolfo, Segovia, Jose C, Samper, Enrique, Surralles, Jordi, Gregory, Philip D, Holmes, Michael C, Naldini, Luigi, Bueren, Juan A
Published 2014

Engineering HIV-Resistant Human CD4+ T Cells with CXCR4-Specific Zinc-Finger Nucleases

by Wilen, Craig B., Wang, Jianbin, Tilton, John C., Miller, Jeffrey C., Kim, Kenneth A., Rebar, Edward J., Sherrill-Mix, Scott A., Patro, Sean C., Secreto, Anthony J., Jordan, Andrea P. O., Lee, Gary, Kahn, Joshua, Aye, Pyone P., Bunnell, Bruce A., Lackner, Andrew A., Hoxie, James A., Danet-Desnoyers, Gwenn A., Bushman, Frederic D., Riley, James L., Gregory, Philip D., June, Carl H., Holmes, Michael C., Doms, Robert W.
Published 2011