Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

per Buchlis, George, Podsakoff, Gregory M., Radu, Antonetta, Hawk, Sarah M., Flake, Alan W., Mingozzi, Federico, High, Katherine A.
Publicat 2012

Impaired Telomere Maintenance and Decreased Canonical WNT Signaling but Normal Ribosome Biogenesis in Induced Pluripotent Stem Cells from X-Linked Dyskeratosis Congenita Patients

per Gu, Bai-Wei, Apicella, Marisa, Mills, Jason, Fan, Jian-Meng, Reeves, Dara A., French, Deborah, Podsakoff, Gregory M., Bessler, Monica, Mason, Philip J.
Publicat a PLoS One (2015)

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

per Kessler, Paul D., Podsakoff, Gregory M., Chen, Xiaojuan, McQuiston, Susan A., Colosi, Peter C., Matelis, Laura A., Kurtzman, Gary J., Byrne, Barry J.
Publicat 1996

Effects of Immunosuppression on Circulating Adeno-Associated Virus Capsid-Specific T cells in Humans

per 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.
Publicat 2013

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

per 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
Publicat a Mol Ther Methods Clin Dev (2015)

Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients

per 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.
Publicat 2013

Single Nucleotide Polymorphism Array Analysis of Bone Marrow Failure Patients Reveals Characteristic Patterns of Genetic Changes

per 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
Publicat 2013

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

per 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.
Publicat 2013

Emergence of Clonal Hematopoiesis in the Majority of Patients with Acquired Aplastic Anemia

per 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
Publicat a Cancer Genet (2015)

Somatic HLA mutations expose the role of class I–mediated autoimmunity in aplastic anemia and its clonal complications

per 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.
Publicat a Blood Adv (2017)

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

per 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.
Publicat 2012