Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
Buchlis, George, Podsakoff, Gregory M., Radu, Antonetta, Hawk, Sarah M., Flake, Alan W., Mingozzi, Federico, High, Katherine A.
Published in Blood (29.03.2012)
Published in Blood (29.03.2012)
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Overcoming preexisting humoral immunity to AAV using capsid decoys
Mingozzi, Federico, Anguela, Xavier M, Pavani, Giulia, Chen, Yifeng, Davidson, Robert J, Hui, Daniel J, Yazicioglu, Mustafa, Elkouby, Liron, Hinderer, Christian J, Faella, Armida, Howard, Carolann, Tai, Alex, Podsakoff, Gregory M, Zhou, Shangzhen, Basner-Tschakarjan, Etiena, Wright, John Fraser, High, Katherine A
Published in Science translational medicine (17.07.2013)
Published in Science translational medicine (17.07.2013)
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Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
Candotti, Fabio, Shaw, Kit L., Muul, Linda, Carbonaro, Denise, Sokolic, Robert, Choi, Christopher, Schurman, Shepherd H., Garabedian, Elizabeth, Kesserwan, Chimene, Jagadeesh, G. Jayashree, Fu, Pei-Yu, Gschweng, Eric, Cooper, Aaron, Tisdale, John F., Weinberg, Kenneth I., Crooks, Gay M., Kapoor, Neena, Shah, Ami, Abdel-Azim, Hisham, Yu, Xiao-Jin, Smogorzewska, Monika, Wayne, Alan S., Rosenblatt, Howard M., Davis, Carla M., Hanson, Celine, Rishi, Radha G., Wang, Xiaoyan, Gjertson, David, Yang, Otto O., Balamurugan, Arumugam, Bauer, Gerhard, Ireland, Joanna A., Engel, Barbara C., Podsakoff, Gregory M., Hershfield, Michael S., Blaese, R. Michael, Parkman, Robertson, Kohn, Donald B.
Published in Blood (01.11.2012)
Published in Blood (01.11.2012)
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Impaired Telomere Maintenance and Decreased Canonical WNT Signaling but Normal Ribosome Biogenesis in Induced Pluripotent Stem Cells from X-Linked Dyskeratosis Congenita Patients
Gu, Bai-Wei, Apicella, Marisa, Mills, Jason, Fan, Jian-Meng, Reeves, Dara A, French, Deborah, Podsakoff, Gregory M, Bessler, Monica, Mason, Philip J
Published in PloS one (18.05.2015)
Published in PloS one (18.05.2015)
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Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency
Reinhardt, Bryanna, Habib, Omar, Shaw, Kit L., Garabedian, Elizabeth, Carbonaro-Sarracino, Denise A., Terrazas, Dayna, Fernandez, Beatriz Campo, De Oliveira, Satiro, Moore, Theodore B., Ikeda, Alan K., Engel, Barbara C., Podsakoff, Gregory M., Hollis, Roger P., Fernandes, Augustine, Jackson, Connie, Shupien, Sally, Mishra, Suparna, Davila, Alejandra, Mottahedeh, Jack, Vitomirov, Andrej, Meng, Wenzhao, Rosenfeld, Aaron M., Roche, Aoife M., Hokama, Pascha, Reddy, Shantan, Everett, John, Wang, Xiaoyan, Luning Prak, Eline T., Cornetta, Kenneth, Hershfield, Michael S., Sokolic, Robert, De Ravin, Suk See, Malech, Harry L., Bushman, Frederic D., Candotti, Fabio, Kohn, Donald B.
Published in Blood (14.10.2021)
Published in Blood (14.10.2021)
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Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients
Garçon, Loïc, Ge, Jingping, Manjunath, Shwetha H., Mills, Jason A., Apicella, Marisa, Parikh, Shefali, Sullivan, Lisa M., Podsakoff, Gregory M., Gadue, Paul, French, Deborah L., Mason, Philip J., Bessler, Monica, Weiss, Mitchell J.
Published in Blood (08.08.2013)
Published in Blood (08.08.2013)
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Somatic HLA mutations expose the role of class I–mediated autoimmunity in aplastic anemia and its clonal complications
Babushok, Daria V., Duke, Jamie L., Xie, Hongbo M., Stanley, Natasha, Atienza, Jamie, Perdigones, Nieves, Nicholas, Peter, Ferriola, Deborah, Li, Yimei, Huang, Hugh, Ye, Wenda, Morrissette, Jennifer J.D., Kearns, Jane, Porter, David L., Podsakoff, Gregory M., Eisenlohr, Laurence C., Biegel, Jaclyn A., Chou, Stella T., Monos, Dimitrios S., Bessler, Monica, Olson, Timothy S.
Published in Blood advances (10.10.2017)
Published in Blood advances (10.10.2017)
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Emergence of clonal hematopoiesis in the majority of patients with acquired aplastic anemia
Babushok, Daria V, Perdigones, Nieves, Perin, Juan C, Olson, Timothy S, Ye, Wenda, Roth, Jacquelyn J, Lind, Curt, Cattier, Carine, Li, Yimei, Hartung, Helge, Paessler, Michele E, Frank, Dale M, Xie, Hongbo M, Cross, Shanna, Cockroft, Joshua D, Podsakoff, Gregory M, Monos, Dimitrios, Biegel, Jaclyn A, Mason, Philip J, Bessler, Monica
Published in Cancer genetics (01.04.2015)
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AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
Hui, Daniel J, Edmonson, Shyrie C, Podsakoff, Gregory M, Pien, Gary C, Ivanciu, Lacramioara, Camire, Rodney M, Ertl, Hildegund, Mingozzi, Federico, High, Katherine A, Basner-Tschakarjan, Etiena
Published in Molecular therapy. Methods & clinical development (01.01.2015)
Published in Molecular therapy. Methods & clinical development (01.01.2015)
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Single nucleotide polymorphism array analysis of bone marrow failure patients reveals characteristic patterns of genetic changes
Babushok, Daria V., Xie, Hongbo M., Roth, Jacquelyn J., Perdigones, Nieves, Olson, Timothy S., Cockroft, Joshua D., Gai, Xiaowu, Perin, Juan C., Li, Yimei, Paessler, Michele E., Hakonarson, Hakon, Podsakoff, Gregory M., Mason, Philip J., Biegel, Jaclyn A., Bessler, Monica
Published in British journal of haematology (01.01.2014)
Published in British journal of haematology (01.01.2014)
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Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein
Kessler, Paul D., Podsakoff, Gregory M., Chen, Xiaojuan, McQuiston, Susan A., Colosi, Peter C., Matelis, Laura A., Kurtzman, Gary J., Byrne, Barry J.
Published in Proceedings of the National Academy of Sciences - PNAS (26.11.1996)
Published in Proceedings of the National Academy of Sciences - PNAS (26.11.1996)
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Clonal evolution and clinical significance of copy number neutral loss of heterozygosity of chromosome arm 6p in acquired aplastic anemia
Betensky, Marisol, Babushok, Daria, Roth, Jacquelyn J, Mason, Philip J, Biegel, Jaclyn A, Busse, Tracy M, Li, Yimei, Lind, Curt, Papazoglou, Anna, Monos, Dimitri, Podsakoff, Gregory, Bessler, Monica, Olson, Timothy S
Published in Cancer genetics (01.01.2016)
Published in Cancer genetics (01.01.2016)
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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
High, Katherine A, Herzog, Roland W, Yang, Edmund Y, Couto, Linda B, Hagstrom, J. Nathan, Elwell, Dan, Fields, Paul A, Burton, Melissa, Bellinger, Dwight A, Read, Marjorie S, Brinkhous, Kenneth M, Podsakoff, Gregory M, Nichols, Timothy C, Kurtzman, Gary J
Published in Nature medicine (01.01.1999)
Published in Nature medicine (01.01.1999)
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Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans
Parzych, Elizabeth M, Li, Hua, Yin, Xiangfan, Liu, Qin, Wu, Te-Lang, Podsakoff, Gregory M, High, Katherine A, Levine, Matthew H, Ertl, Hildegund C J
Published in Human gene therapy (01.04.2013)
Published in Human gene therapy (01.04.2013)
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Ips Cell Lines From Members Of a Family With Variable Penetrance Of An RPS19 Mutation Recapitulate The Diamond Blackfan Anemia (DBA) Phenotype and Show Differences In Ribosome Biogenesis and TGF Beta Signaling
Ge, Jingping, Apicella, Marisa, Manjunath, Shwetha, Mills, Jason A., Garçon, Loïc, Podsakoff, Gregory M., French, Deborah L., Weiss, Mitchell J., Bessler, Monica, Mason, Philip J.
Published in Blood (15.11.2013)
Published in Blood (15.11.2013)
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Abstract 2977: Most patients with acquired aplastic anemia develop clonal hematopoiesis early in disease
Babushok, Daria V., Perdigones, Nieves, Perin, Juan C., Olson, Timothy S., Ye, Wenda, Roth, Jacquelyn J., Lind, Curt, Cattier, Carine, Li, Yimei, Hartung, Helge, Paessler, Michele E., Frank, Dale M., Xie, Hongbo M., Busse, Tracy M., Cross, Shanna, Podsakoff, Gregory M., Monos, Dimitrios, Biegel, Jaclyn A., Mason, Philip J., Bessler, Monica
Published in Cancer research (Chicago, Ill.) (01.08.2015)
Published in Cancer research (Chicago, Ill.) (01.08.2015)
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Single Nucleotide Polymorphism Array Analysis Of Bone Marrow Failure Patients Reveals Characteristic Patterns Of Genetic Changes
Babushok, Daria V., Xie, Hongbo M., Roth, Jacquelyn J., Perdigones, Nieves, Olson, Timothy S., Cockroft, Joshua D., Gai, Xiaowu, Perin, Juan C., Li, Yimei, Paessler, Michele E., Hakonarson, Hakon, Podsakoff, Gregory M., Mason, Philip J., Biegel, Jaclyn A., Bessler, Monica
Published in Blood (15.11.2013)
Published in Blood (15.11.2013)
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A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV
Mingozzi, Federico, Anguela, Xavier M, Pavani, Giulia, Chen, Yifeng, Davidson, Robert J, Hui, Daniel J., Hinderer, Christian J, Faella, Armida, Howard, Crolann, Tai, Alex, Podsakoff, Gregory M, Ragni, Margaret V., Zhou, Shangzhen, Basner-Tschakarjan, Etiena, Wright, J. Fraser, High, Katherine A.
Published in Blood (16.11.2012)
Published in Blood (16.11.2012)
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Impaired Telomere Maintenance and Decreased Canonical WNT Signaling but Normal Ribosome Biogenesis in Induced Pluripotent Stem Cells from X-Linked Dyskeratosis Congenita Patients: e0127414
Gu, Bai-Wei, Apicella, Marisa, Mills, Jason, Fan, Jian-Meng, Reeves, Dara A, French, Deborah, Podsakoff, Gregory M, Bessler, Monica, Mason, Philip J
Published in PloS one (01.05.2015)
Published in PloS one (01.05.2015)
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