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frederic D. Bushman
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Journal Articles

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Maxwell E. Chappell, Laura Breda, Lucas Tricoli, Amaliris Guerra, Danuta Jarocha, Carlo Castruccio Castracani, Tyler E. Papp, Naoto Tanaka, Nolan Hamilton, Michael P. Triebwasser, Valentina Ghiaccio, Megan T. Fedorky, Kandace L. Gollomp, Veronica Bochenek, Aoife M. Roche, John K. Everett, Emma J. Cook, Frederic D. Bushman, Nattiya Teawtrakul, Stavros Glentis, Antonis Kattamis, Barbara L. Mui, Ying K. Tam, Drew Weissman, Osheiza Abdulmalik, Hamideh Parhiz, Stefano Rivella
Journal: Blood
Blood (2024) 144 (15): 1633–1645.
https://doi.org/10.1182/blood.2023023349
Published: 2024
Includes: Supplemental data
Journal Articles

Gene Therapy Expressing Platelet-Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

David A. Wilcox, Myriam Armant, Lynn Malec, Qizhen Shi, Bryon D. Johnson, Huiqing Xu, Lily M. Du, Lisa J. Duffy, Frederic D. Bushman, James H. Jerkins, Peiman Hematti, Ruta Brazauskas, Shawn M. Jobe, Parameswaran N. Hari, Mary Eapen
Journal: Blood
Blood (2023) 142 (Supplement 1): 2249.
https://doi.org/10.1182/blood-2023-172845
Published: 2023
Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Clinical Trials & Observations
Roxane Labrosse, Julia I. Chu, Myriam A. Armant, John K. Everett, Danilo Pellin, Niharika Kareddy, Andrew L. Frelinger, Lauren A. Henderson, Amy E. O’Connell, Amlan Biswas, Jet Coenen-van der Spek, Alexandra Miggelbrink, Claudia Fiorini, Hriju Adhikari, Charles C. Berry, Vito Adrian Cantu, Johnson Fong, Jason Jaroslavsky, Derin F. Karadeniz, Quan-Zhen Li, Shantan Reddy, Aoife M. Roche, Chengsong Zhu, Jennifer S. Whangbo, Colleen Dansereau, Brenda Mackinnon, Emily Morris, Stephanie M. Koo, Wendy B. London, Safa Baris, Ahmet Ozen, Elif Karakoc-Aydiner, Jenny M. Despotovic, Lisa R. Forbes Satter, Akihiko Saitoh, Yuta Aizawa, Alejandra King, Mai Anh Thi Nguyen, Vy Do Uyen Vu, Scott B. Snapper, Anne Galy, Luigi D. Notarangelo, Frederic D. Bushman, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2023) 142 (15): 1281–1296.
https://doi.org/10.1182/blood.2022019117
Published: 2023
Includes: Supplemental data
Journal Articles

Chemotherapy-induced reversal of ciltacabtagene autoleucel–associated movement and neurocognitive toxicity

Clinical Trials & Observations
Charlotte E. Graham, Won-Ho Lee, Hadley R. Wiggin, Valentina M. Supper, Mark B. Leick, Filippo Birocchi, Andrew J. Yee, Angelina Petrichenko, John Everett, Frederic D. Bushman, Hossein Sadrzadeh, Otto Rapalino, Daniel Chiu, Isabel Arrillaga-Romany, Marcela V. Maus, Matthew J. Frigault, Kathleen M. E. Gallagher
Journal: Blood
Blood (2023) 142 (14): 1248–1252.
https://doi.org/10.1182/blood.2023021429
Published: 2023
Includes: Supplemental data
Journal Articles

Novel extragenic genomic safe harbors for precise therapeutic T-cell engineering

Ashlesha Odak, Han Yuan, Judith Feucht, Vito Adrian Cantu, Jorge Mansilla-Soto, Friederike Kogel, Justin Eyquem, John Everett, Frederic D. Bushman, Christina S. Leslie, Michel Sadelain
Journal: Blood
Blood (2023) 141 (22): 2698–2712.
https://doi.org/10.1182/blood.2022018924
Published: 2023
Includes: Supplemental data
Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
Journal Articles

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Claire Booth, Donald B. Kohn, Myriam Armant, Susan Prockop, Karen Buckland, Sharat Chandra, Shanmuganathan Chandrakasan, Kritika Chetty, Colleen Dansereau, Satiro de Oliveira, John Everett, Diego León Rico, Wendy B. London, Jin Hua Xu-Bayford, Theodore B. Moore, Nisha Nagarsheth, Suhag Parikh, Dayna Terrazas, Shanna L White, Axel Schambach, Frederic D. Bushman, Adrian J. Thrasher, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2022) 140 (Supplement 1): 7770–7771.
https://doi.org/10.1182/blood-2022-159979
Published: 2022
Journal Articles

Decade-Long Remissions of Leukemia Sustained By the Persistence of Activated CD4+ CAR T-Cells

J. Joseph Melenhorst, Gregory M Chen, Meng Wang, David L. Porter, Peng Gao, Shovik Bandyopadhyay, Iulian Pruteanu-Malinici, Christopher L Nobles, Sayantan Maji, Noelle V Frey, Saar Gill, Lifeng Tian, Irina Kulikovskaya, Megan M Davis, Joseph A Fraietta, Jennifer L Brogdon, Regina Young, David E Ambrose, Anne Chew, Bruce L Levine, Don L. Siegel, Cécile Alanio, E. John Wherry, Frederic D. Bushman, Simon F Lacey, Kai Tan, Carl H June
Journal: Blood
Blood (2021) 138 (Supplement 1): 166.
https://doi.org/10.1182/blood-2021-152664
Published: 2021
Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Alessandra Magnani, Michaela Semeraro, Frédéric ADAM, Claire Booth, Loic Dupre, Emma C Morris, Aurélie Gabrion, Cecile Roudaut, Delphine Borgel, Antoine Toubert, Emmanuel Clave, Chrystelle Abdo, Guy Gorochov, Rachel Petermann, Mélanie Guiot, Makoto Miyara, Despina Moshous, Elisa Magrin, Adeline Denis, Felipe Suarez, Chantal Lagresle-Peyrou, Aoife Doto, John K. Everett, Amélie Trinquand, Marianne Guisset, Jin Hua Xu-Bayford, Salima Hacein-Bey-Abina, Alexandre Kauskot, Reem Elfeky, Christine Rivat, Sarah Abbas, Bobby H Gaspar, Elizabeth A. Macintyre, Capucine Picard, Frederic D. Bushman, Anne Galy, Alain Fischer, Emmanuelle Six, Adrian J. Thrasher, Marina Cavazzana
Journal: Blood
Blood (2021) 138 (Supplement 1): 2934.
https://doi.org/10.1182/blood-2021-147697
Published: 2021
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

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Emmanuelle Six, Agathe Guilloux, Adeline Denis, Arnaud Lecoules, Alessandra Magnani, Romain Vilette, Frances Male, Nicolas Cagnard, Marianne Delville, Elisa Magrin, Laure Caccavelli, Cécile Roudaut, Clemence Plantier, Steicy Sobrino, John Gregg, Christopher L. Nobles, John K. Everett, Salima Hacein-Bey-Abina, Anne Galy, Alain Fischer, Adrian J. Thrasher, Isabelle André, Marina Cavazzana, Frederic D. Bushman
Journal: Blood
Blood (2020) 135 (15): 1219–1231.
https://doi.org/10.1182/blood.2019002350
Published: 2020
Includes: Supplemental data
Journal Articles

First-in-Human Assessment of Feasibility and Safety of Multiplexed Genetic Engineering of Autologous T Cells Expressing NY-ESO -1 TCR and CRISPR/Cas9 Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T cells) in Advanced Multiple Myeloma (MM) and Sarcoma

Edward A. Stadtmauer, MD FACP, Adam D. Cohen, MD, Kristy Weber, MD, Simon F Lacey, PhD, Vanessa E. Gonzalez, J. Joseph Melenhorst, PhD, Joseph A Fraietta, PhD, Gabriela Plesa, MD, Joanne Shea, Tina Matlawski, RN, Amanda Cervini, RN, Patricia Mangan, CRNP, Avery Gaymon, Stephanie Desjardins, Eric Lancaster, MD, January Salas-Mckee, Megan M. Suhoski, PhD, Andrew Fesnak, MD, Matthew O'Rourke, Anne Lamontagne, Don L. Siegel, MD PhD, Regina M Young, PhD, Anne Chew, PhD, Christopher L Nobles, PhD, Frederic D. Bushman, PhD, Howard Y. Chang, MD PhD, Ansuman T. Satpathy, MD PhD, Yangbing Zhao, PhD MD, Wei-Ting Hwang, PhD, Elizabeth O. Hexner, MDMS, Carl H June, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 49.
https://doi.org/10.1182/blood-2019-122374
Published: 2019
Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 611.
https://doi.org/10.1182/blood-2019-126007
Published: 2019
Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Roxane Labrosse, MD FRCPC, Julia Chu, MD MPH, Myriam Armant, PhD, Jet van der Spek, MD, Alexandra Miggelbrink, MD, Johnson Fong, John K. Everett, PhD, Hayley Raymond, BA, Lyanna Kessler, Colleen Dansereau, MSN, RN, CPN, Brenda Mackinnon, BSN, RN, Stephanie Koo, Emily Morris, Wendy B. London, PhD, Ahmet Ozen, MD, Safa Baris, MD, Jenny M. Despotovic, DO, MS, Lisa Forbes, MD, Akihiko Saitoh, MD, Takayuki Takachi, MD, Alejandra King, MD, Thi Mai Anh Thi Nguyen, MD, Vy Do Uyen Vu, MD, Frederic D. Bushman, PhD, Anne Galy, PharmD, PhD, Luigi Notarangelo, MD, David A Williams, MD, Sung-Yun Pai, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4629.
https://doi.org/10.1182/blood-2019-126161
Published: 2019
Journal Articles

Long-Term Remission of CLL Sustained By Pauciclonal Anti-CD19 Chimeric Antigen Receptor T (CTL019) Cell Clones

J. Joseph Melenhorst, PhD, David L. Porter, MD, Lifeng Tian, PhD, Simon F Lacey, PhD BS, Christopher L Nobles, PhD, Joseph A Fraietta, PhD, Noelle V. Frey, MD, Irina Kulikovskaya, Minnal Gupta, Regina M Young, PhD, David E Ambrose, BS, Don L. Siegel, MD PhD, Frederic D Bushman, PhD, Carl H June, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 699.
https://doi.org/10.1182/blood-2018-99-117390
Published: 2018
Journal Articles

Vector Integration and Efficacy of CD19-Directed CAR T Cell Therapy in Acute Lymphoblastic Leukemia (ALL) and Chronic Lymphocytic Leukemia (CLL)

Christopher L Nobles, PhD, John K Everett, Shantan Reddy, Joseph A Fraietta, PhD, David L. Porter, MD, Noelle V. Frey, MD, Stephan A. Grupp, MD PhD, Don L. Siegel, MD PhD, Simon F Lacey, PhD BS, Carl H June, MD, Frederic D Bushman, PhD, J. Joseph Melenhorst, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4548.
https://doi.org/10.1182/blood-2018-99-117034
Published: 2018
Journal Articles

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Punam Malik, MD, Michael Grimley, MD, Charles T. Quinn, MD, Amy Shova, RN, Little Courtney, RN, Carolyn Lutzko, PhD, Theodosia A. Kalfa, MD PhD, Omar Niss, MD, Parinda A Mehta, MD, Sharat Chandra, MBBS,MD, Elke Grassman, Johannes C.M. Van der Loo, PhD, Scott Witting, MS, Diana Nordling, Archana Shreshta, PhD, Sydney Felker, BS, Catherine Terrell, BS, Lilith Reeves, MS, MT(ASCP), Devin Pillis, MS, Loberg Anastacia, Frederic D Bushman, PhD, Jennifer Knight-Madden, Karen Kalinyak, MD, Stella M. Davies, PhD MBBS, MRCP, Monika Asnani, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1021.
https://doi.org/10.1182/blood-2018-99-119591
Published: 2018
Journal Articles

Clinical Evidence That Deficiency of the Methylcytosine Dioxygenase TET2 Gene Promotes the Clonal Expansion and Therapeutic Efficacy of CD19-Directed T Cells

Joseph A Fraietta, PhD, Christopher L Nobles, PhD, Morgan A Sammons, PhD, Shannon A. Carty, MD, Stefan M Lundh, BS, Tyler Reich, Alexandria P Cogdill, BS, MB, Mercy Gohil, Jennifer J.D. Morrissette, PhD, Irina Kulikovskaya, Farzana Nazimuddin, MS, Minnal Gupta, Yan Wang, PhD, Xiaojun Liu, Regina M Young, PhD, David E Ambrose, Martha S Jordan, PhD, Katherine T Marcucci, PhD, Bruce L Levine, PhD, Yangbing Zhao, PhD MD, Michael Kalos, PhD, David L Porter, MD, Simon F Lacey, PhD BS, Shelley L Berger, PhD BS, Frederic D Bushman, PhD, Carl H June, MD, J. Joseph Melenhorst, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1909.
https://doi.org/10.1182/blood.V130.Suppl_1.1909.1909
Published: 2017
Journal Articles

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Emmanuelle Six, Agathe Guilloux, Christopher Nobles, Rawya Zreik, Arnaud Lecoules, John Everett, Romain Vilette, Nicolas Cagnard, PhD, Frances Male, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Caccavelli, Cecile Roudaut, Clemence Plantier, Anne Galy, PharmD, PhD, Adrian J Thrasher, MD PhD FMedSci, Isabelle Andre-Schmutz, Charles C Berry, Marina Cavazzana, MD PhD, Frederic D Bushman, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4618.
https://doi.org/10.1182/blood.V130.Suppl_1.4618.4618
Published: 2017
Journal Articles

Cars in Leukemia: Relapse with Antigen-Negative Leukemia Originating from a Single B Cell Expressing the Leukemia-Targeting CAR

Simon Francis Lacey, PhD BS, Jun Xu, PhD, Marco Ruella, MD, David M. Barrett, MD PhD, Irina Kulikovskaya, PhD, David E Ambrose, BS, Prachi R. Patel, BS, Tyler Reich, John Scholler, Farzana Nazimuddin, Joseph A. Fraietta, PhD, Shannon L Maude, MD PhD, Saar I. Gill, MD PhD, Bruce L Levine, PhD, Christopher L Nobles, PhD, Frederic D Bushman, PhD, Elena Orlando, PhD, Stephan A. Grupp, MD PhD, Carl H June, MD, Jan Joseph Melenhorst, PhD
Journal: Blood
Blood (2016) 128 (22): 281.
https://doi.org/10.1182/blood.V128.22.281.281
Published: 2016