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

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

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

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

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

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

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

gan 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.
Cyhoeddwyd 2013

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

gan 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
Cyhoeddwyd yn Mol Ther Methods Clin Dev (2015)

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

gan 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.
Cyhoeddwyd 2013

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

gan 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
Cyhoeddwyd 2013

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

gan 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.
Cyhoeddwyd 2013

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

gan 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
Cyhoeddwyd yn Cancer Genet (2015)

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

gan 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.
Cyhoeddwyd yn Blood Adv (2017)

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

gan 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.
Cyhoeddwyd 2012