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Gregory M Podsakoff
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Journal Articles

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Clinical Trials & Observations
Bryanna Reinhardt, Omar Habib, Kit L. Shaw, Elizabeth Garabedian, Denise A. Carbonaro-Sarracino, Dayna Terrazas, Beatriz Campo Fernandez, Satiro De Oliveira, Theodore B. Moore, Alan K. Ikeda, Barbara C. Engel, Gregory M. Podsakoff, Roger P. Hollis, Augustine Fernandes, Connie Jackson, Sally Shupien, Suparna Mishra, Alejandra Davila, Jack Mottahedeh, Andrej Vitomirov, Wenzhao Meng, Aaron M. Rosenfeld, Aoife M. Roche, Pascha Hokama, Shantan Reddy, John Everett, Xiaoyan Wang, Eline T. Luning Prak, Kenneth Cornetta, Michael S. Hershfield, Robert Sokolic, Suk See De Ravin, Harry L. Malech, Frederic D. Bushman, Fabio Candotti, Donald B. Kohn
Journal: Blood
Blood (2021) 138 (15): 1304–1316.
https://doi.org/10.1182/blood.2020010260
Published: 2021
Includes: Supplemental data
Journal Articles

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

Daria V. Babushok, Hongbo M. Xie, Jacquelyn J. Roth, Nieves Perdigones, Timothy S. Olson, Joshua D. Cockroft, Xiaowu Gai, Juan C. Perin, Yimei Li, Michele E. Paessler, Hakon Hakonarson, Gregory M. Podsakoff, Philip J. Mason, Jaclyn A. Biegel, Monica Bessler
Journal: Blood
Blood (2013) 122 (21): 3710.
https://doi.org/10.1182/blood.V122.21.3710.3710
Published: 2013
Journal Articles

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

Jingping Ge, Marisa Apicella, Shwetha Manjunath, Jason A. Mills, Loïc Garçon, Gregory M. Podsakoff, Deborah L. French, Mitchell J. Weiss, Monica Bessler, Philip J. Mason
Journal: Blood
Blood (2013) 122 (21): 1224.
https://doi.org/10.1182/blood.V122.21.1224.1224
Published: 2013
Journal Articles

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

Loïc Garçon, Jingping Ge, Shwetha H. Manjunath, Jason A. Mills, Marisa Apicella, Shefali Parikh, Lisa M. Sullivan, Gregory M. Podsakoff, Paul Gadue, Deborah L. French, Philip J. Mason, Monica Bessler, Mitchell J. Weiss
Journal: Blood
Blood (2013) 122 (6): 912–921.
https://doi.org/10.1182/blood-2013-01-478321
Published: 2013
Includes: Supplemental data
Journal Articles

A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV

Federico Mingozzi, PhD, Xavier M Anguela, PhD, Giulia Pavani, MS, Yifeng Chen, MD, Robert J Davidson, Daniel J. Hui, Ph.D., Christian J Hinderer, MS, Armida Faella, Crolann Howard, BS, Alex Tai, MS, Gregory M Podsakoff, Margaret V. Ragni, MD, MPH, Shangzhen Zhou, MD, Etiena Basner-Tschakarjan, J. Fraser Wright, PhD, Katherine A. High, MD
Journal: Blood
Blood (2012) 120 (21): 2050.
https://doi.org/10.1182/blood.V120.21.2050.2050
Published: 2012
Journal Articles

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

Clinical Trials & Observations
Fabio Candotti, Kit L. Shaw, Linda Muul, Denise Carbonaro, Robert Sokolic, Christopher Choi, Shepherd H. Schurman, Elizabeth Garabedian, Chimene Kesserwan, G. Jayashree Jagadeesh, Pei-Yu Fu, Eric Gschweng, Aaron Cooper, John F. Tisdale, Kenneth I. Weinberg, Gay M. Crooks, Neena Kapoor, Ami Shah, Hisham Abdel-Azim, Xiao-Jin Yu, Monika Smogorzewska, Alan S. Wayne, Howard M. Rosenblatt, Carla M. Davis, Celine Hanson, Radha G. Rishi, Xiaoyan Wang, David Gjertson, Otto O. Yang, Arumugam Balamurugan, Gerhard Bauer, Joanna A. Ireland, Barbara C. Engel, Gregory M. Podsakoff, Michael S. Hershfield, R. Michael Blaese, Robertson Parkman, Donald B. Kohn
Journal: Blood
Blood (2012) 120 (18): 3635–3646.
https://doi.org/10.1182/blood-2012-02-400937
Published: 2012
Includes: Supplemental data
Journal Articles

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

Brief Report
George Buchlis, Gregory M. Podsakoff, Antonetta Radu, Sarah M. Hawk, Alan W. Flake, Federico Mingozzi, Katherine A. High
Journal: Blood
Blood (2012) 119 (13): 3038–3041.
https://doi.org/10.1182/blood-2011-09-382317
Published: 2012