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Frederic Bushman, PhD
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Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Salima Hacein-Bey-Abina, Pharm.D., Ph.D., Sung-Yun Pai, MD, Frederic Bushman, PhD, Myriam Armant, PhD, Stephane Blanche, MD, Johanna Blondeau, Laure Caccavelli, Heather Daley, BS, Colleen Dansereau, RN, BSN, CPN, CPHON, Satiro deOliveira, MD, Dongjing Guo, MPH, Gregory Hopkins, BS, Annick Lim, M.Sci, Wendy B. London, PHD, Nirav Malani, Frances Male, BA, Punam Malik, MD, Maria Angelica Marinovic, MD, Sean McDonough, MS, Matias Oleastro, MD, Capucine Picard, MD PhD, Jerome Ritz, MD, Axel Schambach, MD PhD, Leslie E Silberstein, MD, Alla Tsytsykova, PhD, Johannes C.M. Van der Loo, PhD, Christopher Baum, MD, H. Bobby Gaspar, MD PhD, Donald B. Kohn, MD, Alexandra H. Filipovich, MD, Luigi Daniele Notarangelo, MD, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2013) 122 (21): 715.
https://doi.org/10.1182/blood.V122.21.715.715
Published: 2013
Journal Articles

Genomic Safe Harbors in Human iPS Cells

Eirini P. Papapetrou, MD, PhD, Michel Sadelain, MD, PhD, Frederic Bushman, PhD
Journal: Blood
Blood (2011) 118 (21): SCI-47.
https://doi.org/10.1182/blood.V118.21.SCI-47.SCI-47
Published: 2011
Journal Articles

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

Jennifer E Adair, PhD, Brian C Beard, PhD, Grant Trobridge, PhD, Frederic Bushman, PhD, Maciej M Mrugala, MD PhD, Hans-Peter Kiem, MD
Journal: Blood
Blood (2011) 118 (21): 2053.
https://doi.org/10.1182/blood.V118.21.2053.2053
Published: 2011
Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Sung-Yun Pai, MD, Luigi Daniele Notarangelo, MD, Chad Harris, Federica Cattaneo, MD, Matthew Wladkowski, Myriam Armant, PhD, Grace S. Kao, MD, Matthew Wielgosz, Arthur W. Nienhuis, MD, Ute Modlich, DVM, PhD, Martijn Brugman, PhD, Axel Schambach, MD PhD, Michael Rothe, Susannah I Thornhill, PhD, H. Bobby Gaspar, Kathryn Parsley, PhD, Samantha Cooray, PhD, Elke Grassman, Ph.D., Frederic Bushman, PhD, Sally Shupien, Matias Oleastro, MD, Salima Hacein-Bey-Abina, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, Punam Malik, MD, Alexandra Filipovich, MD, Donald B. Kohn, MD, Christopher Baum, MD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2011) 118 (21): 164.
https://doi.org/10.1182/blood.V118.21.164.164
Published: 2011
Journal Articles

Therapeutic Transgene Expression From Genomic Safe Harbors In Patient-Specific Induced Pluripotent Stem Cells

Eirini P Papapetrou, MD, PhD, Gabsang Lee, PhD, Nirav Malani, Manu Setty, Isabelle Riviere, PhD, Laxmi MS Tirunagari, Kyuichi Kadota, MD, Shoshannah L Roth, PhD, Patricia Giardina, MD, Agnes Viale, PhD, Christina Leslie, PhD, Frederic Bushman, PhD, Lorenz Studer, MD, Michel Sadelain, MD, PhD
Journal: Blood
Blood (2010) 116 (21): 564.
https://doi.org/10.1182/blood.V116.21.564.564
Published: 2010