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Journal Articles

Dissecting the Immune Cell Progeny of CAR-Expressing Hematopoietic Stem Cells in a Humanized Mouse Model

Heng-Yi Liu, Nezia Rahman, Tzu-Ting Chiou, PhD, Satiro N. De Oliveira, MD

Blood (2019) 134 (Supplement_1): 4640.

https://doi.org/10.1182/blood-2019-129746

Published: 2019

Journal Articles

NUP98-KDM5A Fusion Induces Hematopoietic Cell Proliferation and Alters Myelo-Erythropoietic Differentiation

Jonathan Miller, MD PhD, Ryan Hiltenbrand, MS, Tamara Lamprecht, BS, Aman Seth, MS, Sherif Abdelhamed, PhD, Ilaria Iacobucci, PhD, Charles G. Mullighan, MBBS, MD, Jeffery M. Klco, MD PhD

Blood (2019) 134 (Supplement_1): 3775.

https://doi.org/10.1182/blood-2019-130768

Published: 2019

Journal Articles

Targeting WNT10B-Driven Signalling through Induction of FZD6 By Porcupine Inhibition in T Cell Acute Lymphoblastic Leukemia

Adriana Cassaro, PhD, Francesca Lazzaroni, Dr, Giovanni Grillo, MD, Gianluigi Reda, MD, Roberto Cairoli, MD, Alessandro Beghini

Blood (2019) 134 (Supplement_1): 3956.

https://doi.org/10.1182/blood-2019-124871

Published: 2019

Journal Articles

F-Deletion Non-Integrating Measles Virus Vector: A Promising Tool for T-Cell Engineering and Naive iPSCs Generation

Jiyuan Liao, PhD, Yasushi Soda, MD PhD, Ai Sugawara, B.Sc., Yoshie Miura, PhD, Takafumi Hiramoto, PhD, Maino Tahara, PhD, Yuto Takishima, M.S., Shohei Miyamoto, PhD, Makoto Takeda, MD PhD, Kenzaburo Tani, MD PhD

Blood (2019) 134 (Supplement_1): 5750.

https://doi.org/10.1182/blood-2019-131593

Published: 2019

Journal Articles

IPO11 Is Upregulated in Relapsed AML and Supports Survival of Leukemic Stem Cells

Boaz Nachmias, MD PhD, Veronique Voisin, PhD, Rose Hurren, BSc, Xioaming Wang, Neil Maclean, G. Wei Xu, Ayesh K Seneviratne, BSc, MSc, Changjiang Xu, Gary D Bader, Liran I. Shlush, MD, Aaron D Schimmer, MD PhD

Blood (2019) 134 (Supplement_1): 2530.

https://doi.org/10.1182/blood-2019-122284

Published: 2019

Journal Articles

Large-Scale Manufacturing of CMV-CD19CAR T Cells and Characterization of Their Biologic and Immunologic Properties

Xiuli Wang, PhD, Miriam Walter, Ryan Urak, Laura Lim, Vibhuti Vyas, MS, Sally Mokhtari, PhD, Don J. Diamond, PhD, Angelo Cardoso, PhD, John Zaia, MD, Stephen J. Forman, MD, Ryotaro Nakamura, MD

Blood (2019) 134 (Supplement_1): 3247.

https://doi.org/10.1182/blood-2019-131843

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

A Stemness Screen Reveals C3ORF54/INKA1 As a Gate-Keeper of Human Stem Cell Latency

Kerstin B. Kaufmann, PhD, Laura Garcia Prat, Shin-Ichiro Takayanagi, PhD, Jessica McLeod, BSc, Olga I. Gan, Kristele Pan, Stephanie Zhi-Juan Xie, PhD, John E. Dick, PhD

Blood (2018) 132 (Supplement 1): 325.

https://doi.org/10.1182/blood-2018-99-115065

Published: 2018

Journal Articles

Improved Lentiviral Vectors for the Cure of Hemoglobinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Hanyia Zaidi, Silvia Pires Lourenco, MSc, Carla Casu, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD, Vanessa Carrion, RN, CPN, Virginia Guzikowski, RN, MSN, CRNP, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Stefano Rivella, PhD

Blood (2018) 132 (Supplement 1): 3477.

https://doi.org/10.1182/blood-2018-99-111319

Published: 2018

Journal Articles

Suppression of CUL4B Induces G1 Cell Cycle Arrest through the Modulation of AKT Signaling Pathway

Ying Li, MD, Ya Zhang, MD, Xiangxiang Zhou, MD, Xin Wang, MDPhD

Blood (2018) 132 (Supplement 1): 5133.

https://doi.org/10.1182/blood-2018-99-118442

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

Blood (2018) 132 (Supplement 1): 1021.

https://doi.org/10.1182/blood-2018-99-119591

Published: 2018

Journal Articles

Activation of CRLF2/IL7RA Signaling in Normal Human Cord Blood Hematopoietic Progenitors Induces Philadelphia-like B-Cell Precursor Pre-Leukemia and Leukemia In Vivo

Ifat Geron, PhD, Angela Maria Savino, PhD, Noa Tal, MD PhD, John Brown, PhD, Virginia A Turati, DPhil, Yu Nee Lee, PhD, Chela James, PhD, Avigail Rein Gil, MD, Yehudit Birger, PhD, Inna Muler, Michal Hameiri, PhD, Victoria Marcu-Malina, PhD, Ute Fischer, PhD, Arndt Borkhardt, MD, Ilan M. Kirsch, MD, Arnon Nagler, MD, Tariq Enver, PhD, Shai Izraeli, MD

Blood (2018) 132 (Supplement 1): 566.

https://doi.org/10.1182/blood-2018-99-115913

Published: 2018

Journal Articles

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

Henrik Sperber, PhD, Shailee Vora, BS, MS, Andriana G. Kotini, PhD, Malgorzata Olszewska, MS, Aleksandra Wroblewska, PhD, Brian Brown, PhD, Eirini P. Papapetrou, MD PhD

Blood (2018) 132 (Supplement 1): 3083.

https://doi.org/10.1182/blood-2018-99-111857

Published: 2018

Journal Articles

Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections

Blood (2018) 131 (5): 533–545.

https://doi.org/10.1182/blood-2017-10-812859

Published: 2018

Includes: Multimedia, Supplemental data

Journal Articles

Successful Plerixafor-Mediated Mobilization, Apheresis, and Lentiviral Vector Transduction of Hematopoietic Stem Cells in Patients with Severe Sickle Cell Disease

John F Tisdale, MD, Francis J. Pierciey, Jr., Rammurti Kamble, MD, Julie Kanter, MD, Lakshmanan Krishnamurti, MD, Janet L. Kwiatkowski, MD, Alexis A Thompson, MD MPH, Ilya Shestopalov, PhD, Melissa Bonner, PhD, Marcelyne Joseney-Antoine, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2017) 130 (Supplement 1): 990.

https://doi.org/10.1182/blood.V130.Suppl_1.990.990

Published: 2017

Journal Articles

CARM1/PRMT4 As a Novel Therapeutic Target for AML

Sarah Greenblatt, PhD, Na Man, Pierre-Jacques Hamard, PhD, Concepcion Martinez, B.S., Ye Xu, PhD, Fan Liu, PhD, Justin M Watts, MD, Daniel G. Tenen, MD, Stephen D. Nimer, MD

Blood (2017) 130 (Supplement 1): 241.

https://doi.org/10.1182/blood.V130.Suppl_1.241.241

Published: 2017

Journal Articles

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

Blood (2017) 130 (Supplement 1): 523.

https://doi.org/10.1182/blood.V130.Suppl_1.523.523

Published: 2017

Journal Articles

Development of a Treg Lineage Specific Lentiviral Vector for the Treatment of IPEX Syndrome

Katelyn E Masiuk, BS, Jennifer Laborada, BS, Roger P Hollis, PhD, Donald B Kohn, MD

Blood (2017) 130 (Supplement 1): 528.

https://doi.org/10.1182/blood.V130.Suppl_1.528.528

Published: 2017

Journal Articles

Automated Manufacture of CAR19 Donor Lymphocytes Incorporated into an Escalated Dose DLI Protocol: Preliminary Results from the CARD Study

Blood (2017) 130 (Supplement 1): 1993.

https://doi.org/10.1182/blood.V130.Suppl_1.1993.1993

Published: 2017

Journal Articles

Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors

Alessio Cantore, Michela Milani, Andrea Annoni, Tongyao Liu, Sara Bartolaccini, Mauro Biffi, Fabio Russo, Robert Peters, PhD, Angelo Lombardo, Timothy C. Nichols, MD, Eduard Ayuso, Luigi Naldini

Blood (2017) 130 (Supplement 1): 605.

https://doi.org/10.1182/blood.V130.Suppl_1.605.605

Published: 2017

NARROW

Dissecting the Immune Cell Progeny of CAR-Expressing Hematopoietic Stem Cells in a Humanized Mouse Model

NUP98-KDM5A Fusion Induces Hematopoietic Cell Proliferation and Alters Myelo-Erythropoietic Differentiation

Targeting WNT10B-Driven Signalling through Induction of FZD6 By Porcupine Inhibition in T Cell Acute Lymphoblastic Leukemia

F-Deletion Non-Integrating Measles Virus Vector: A Promising Tool for T-Cell Engineering and Naive iPSCs Generation

IPO11 Is Upregulated in Relapsed AML and Supports Survival of Leukemic Stem Cells

Large-Scale Manufacturing of CMV-CD19CAR T Cells and Characterization of Their Biologic and Immunologic Properties

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

A Stemness Screen Reveals C3ORF54/INKA1 As a Gate-Keeper of Human Stem Cell Latency

Improved Lentiviral Vectors for the Cure of Hemoglobinopathies

Suppression of CUL4B Induces G1 Cell Cycle Arrest through the Modulation of AKT Signaling Pathway

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

Activation of CRLF2/IL7RA Signaling in Normal Human Cord Blood Hematopoietic Progenitors Induces Philadelphia-like B-Cell Precursor Pre-Leukemia and Leukemia In Vivo

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections

Successful Plerixafor-Mediated Mobilization, Apheresis, and Lentiviral Vector Transduction of Hematopoietic Stem Cells in Patients with Severe Sickle Cell Disease

CARM1/PRMT4 As a Novel Therapeutic Target for AML

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

Development of a Treg Lineage Specific Lentiviral Vector for the Treatment of IPEX Syndrome

Automated Manufacture of CAR19 Donor Lymphocytes Incorporated into an Escalated Dose DLI Protocol: Preliminary Results from the CARD Study

Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors

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American Society of Hematology

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American Society of Hematology

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