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Frederic D Bushman, PhD
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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

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

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

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

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

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

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

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

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
Journal Articles

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Julia I Chu, MD MPH, Lauren A Henderson, MD, Myriam Armant, PhD, Frances Male, BA, Colleen H Dansereau, MSN, RN, Brenda MacKinnon, RN, Christopher J Burke, Matthew E Cavanaugh, Wendy B. London, PhD, Isil B Barlan, MD, Ahmet Özen, MD, Safa Baris, MD, Elif Karakoc-Aydiner, MD, Ayça Kiykim, MD, Imelda Celine Hanson, MD, Jenny M. Despotovic, DO, MS, Akihiko Saitoh, MD PhD, Takayuki Takachi, MD PhD, Kohsuke Imai, MD PhD, Alejandra King, MD, Silvia Arredondo, MD, Jerome Ritz, MD, Frederic D Bushman, PhD, Anne Galy, PharmD, PhD, Luigi Daniele Notarangelo, MD, David A. Williams, MD, Sung-Yun Pai, MD
Journal: Blood
Blood (2015) 126 (23): 260.
https://doi.org/10.1182/blood.V126.23.260.260
Published: 2015
Journal Articles

Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice.

Hojun Li, Nirav Malani, Shari R Hamilton, DVM, Alexander Schlachterman, MD, Jim Z Zhang, PhD, Giulio Bussadori, Shyrie A Edmonson, Federico Mingozzi, PhD, J. Fraser Wright, PhD, Valder R. Arruda, MD, PhD, Frederic D Bushman, PhD, Katherine A. High, MD
Journal: Blood
Blood (2009) 114 (22): 2465.
https://doi.org/10.1182/blood.V114.22.2465.2465
Published: 2009