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

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

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

Por Gu, Bai-Wei, Apicella, Marisa, Mills, Jason, Fan, Jian-Meng, Reeves, Dara A., French, Deborah, Podsakoff, Gregory M., Bessler, Monica, Mason, Philip J.
Publicado no PLoS One (2015)

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

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

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

Por 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.
Publicado em 2013

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

Por 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
Publicado no Mol Ther Methods Clin Dev (2015)

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

Por 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.
Publicado em 2013

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

Por 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
Publicado em 2013

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

Por 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.
Publicado em 2013

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

Por 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
Publicado no Cancer Genet (2015)

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

Por 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.
Publicado no Blood Adv (2017)

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

Por 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.
Publicado em 2012