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Frederic Bushman
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Journal Articles

CCR5 blockade: A therapeutic approach to uncouple CART-BCMA expansion from CART-mediated immune toxicities.

Free
Luca Paruzzo, Matthew Ho, Julia Han Noll, Federico Stella, Ivan Cohen, Angel Ramirez-Fernandez, Adam Waxman, Shivani Kapur, Fang Chen, Andrew Huff, Danuta Jarocha, Owen Koucky, Caitlin Hopkins, Alex Dimitri, Maya Lavorando, Dan Vogl, John Scholler, Aoife Doto, John Everett, Frederic Bushman, Katherine Nathanson, Patrizia Porazzi, Edward Stadtmauer, Sandra Susanibar-Adaniya, Alfred Garfall, Adam Cohen, Joseph Fraietta, Marco Ruella
Journal: Blood
Blood (2025) 146 (Supplement 1): 5945.
https://doi.org/10.1182/blood-2025-5945
Published: 2025
Journal Articles

Ruxolitinib for refractory immune effector cell enterocolitis following ciltacabtagene autoleucel CAR T-cell therapy for multiple myeloma

Free
Viktoria Blumenberg, Filippo Birocchi, Angela Shih, Giulia Escobar, Adele Mucci, Alexander Armstrong, Diana Cirstea, Deshea L Harris, David Bozym, Benjamin Puliafito, Richard Newcomb, Tejaswini Dhawale, Patrick Connor Johnson, Areej El-Jawahri, Richard Jeffrey, Alexis Barselau, Alex Li, Estelle Emmanuel-Alejandro, Daniella Cook, Kevin Lindell, Samantha Luk, Ryan Chaffee, Andrew Yee, Andrew Branagan, Noopur Raje, Charlotte Graham, Sarah Hammond, Frederic Bushman, Aoife Doto, Shanyan Reddy, Alexander McFarland, Yi-Bin Chen, Christopher Mount, Michael Dougan, Valentina Nardi, Aliyah Sohani, Kathleen ME Gallagher, Marcela Maus, Matthew Frigault, Mark Leick
Journal: Blood
Blood (2025) 146 (Supplement 1): 2392.
https://doi.org/10.1182/blood-2025-2392
Published: 2025
Journal Articles

Patient-informed CRISPR screening reveals gene targets to improve CAR T-cell efficacy

Free
Beatriz Coutinho De Oliveira, Meraj Khan, Cassandra Gilmour, Saaurav Bari, Jessica Kerr, Yee Peng Phoon, Joseph Fraietta, Frederic Bushman, J. Joseph Melenhorst
Journal: Blood
Blood (2025) 146 (Supplement 1): 106.
https://doi.org/10.1182/blood-2025-106
Published: 2025
Journal Articles

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Free
Erica Esrick, Leslie Lehmann, Amy Federico, Heather Daley, Colleen Dansereau, Satiro DeOliveira, John Everett, Pei-Chi Kao, Boya Liu, Theodore Moore, Emily Morris, Helene Negre, Kit Shaw, Gavin Roach, Jerome Ritz, Aoife Doto, Olivia Silva, Helene Vincon, Frederic Bushman, Donald Kohn, Akshat Jain, Wendy London, David Justus, Myriam Armant, John Manis, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 2533.
https://doi.org/10.1182/blood-2025-2533
Published: 2025
Journal Articles

Delayed neurotoxicity, enterocolitis, and BCMA-CART-associated immune-related adverse events (CirAE)  are caused by CD4+ CAR T-cells

Free
Julia Han Noll, Matthew Ho, Luca Paruzzo, Federico Stella, Pooja Devi, Sonia Ndeupen, Gregory Chen, Ivan Cohen, Angel Ramirez-Fernandez, Adam Waxman, Shivani Kapur, Fang Chen, Rong Xu, Andrew Huff, Danuta Jarocha, Vrutti Patel, Audrey Bochi-Layec, Ranjani Ramasubramanian, Shan Liu, Riemke Bouvier, Vitor Souza, Heta Patel, Ziyu Li, Alberto Carturan, Peter Michener, Caitlin Hopkins, Owen Koucky, Janna Minehart, Alex Dimitri, Neel Nabar, Zainul Hasanali, Bryan Ciccarelli, Erik Williams, Robert Bartoszek, Maya Lavorando, Suyash Mohan, Vanessa Gonzalez, Patrizia Porazzi, Vijay Bhoj, Sokratis Apostolidis, Dan Vogl, David Porter, John Scholler, Caroline Diorio, Aoife Doto, John Everett, Frederic Bushman, Katherine Nathanson, Edward Stadtmauer, Sandra Susanibar-Adaniya, Alfred Garfall, Marco Ruella, Adam Cohen, Joseph Fraietta
Journal: Blood
Blood (2025) 146 (Supplement 1): 804.
https://doi.org/10.1182/blood-2025-804
Published: 2025
Journal Articles

Toward ‘n of one’ gene therapies: Scalable ex vivo adenine base editing via lipid nanoparticles in a sickle cell disease model

Free
Sarah Cotteta, Omar Banda, Friederike Herbst-Nowrouzi, Brooklyn McDonald, Yongping Wang, Mohamad-Gabriel Alameh, Frederic Bushman, Stephan Grupp, Eugene Khandros, Stephan Kadauke
Journal: Blood
Blood (2025) 146 (Supplement 1): 7835.
https://doi.org/10.1182/blood-2025-7835
Published: 2025
Journal Articles

Dual-Vector-Transduction with Single-Step Magnetic Selection (Zip-sort) Increases Transgenic Payloads to Enhance CAR T Cell Activity

Free
Scott E. James, Sophia Chen, Brandon Ng, Jacob Fischman, Lorenz Jahn, Alexander P. Boardman, Adhithi Rajagopalan, Harold K Elias, Alyssa R Massa, Dylan Manuele, Katherine Nichols, Amina Lazrak, Nicole Lee, Aoife Doto, Alexander McMarland, Angelina Petrichenko, John Everett, Frederic Bushman, Teng Fei, Anastasia Kousa, Andri Leo L Lemarquis, Susan DeWolf, Jonathan U. Peled, Santosha A. Vardhana, Christopher A. Klebanoff, Marcel R.M. van den Brink
Journal: Blood
Blood (2024) 144 (Supplement 1): 501.
https://doi.org/10.1182/blood-2024-208357
Published: 2024
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

Free
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

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

Free
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

Genomic Safe Harbors in Human iPS Cells

Free
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

Free
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

Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

Free
Olivier Negre, Floriane Fusil, Charlotte Colomb, Shoshannah Roth, Beatrix Gillet-Legrand, Annie Henri, Yves Beuzard, Frederic Bushman, Philippe Leboulch, Emmanuel Payen
Journal: Blood
Blood (2011) 117 (20): 5321–5331.
https://doi.org/10.1182/blood-2010-01-263582
Published: 2011
Includes: Supplemental data
Journal Articles

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

Free
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