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Journal Articles

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Richard J. Jones, Michael R. DeBaun

Blood (2021) 138 (11): 942–947.

https://doi.org/10.1182/blood.2021011488

Published: 2021

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

Homeobox Transcription Factor Hhex Promotes Myeloid Leukemia in Cooperation with Mutant ASXL1

Blood (2019) 134 (Supplement_1): 2525.

https://doi.org/10.1182/blood-2019-126663

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

Blood (2018) 132 (Supplement 1): 4548.

https://doi.org/10.1182/blood-2018-99-117034

Published: 2018

Journal Articles

Synergy between FLT3-ITD and p53 Haploinsufficiency or Loss in the Development of Acute Myeloid Leukemia

Zengkai Pan, MD PhD, Min Yang, Kezhi Huang, MD PhD, Guntram Buesche, MD, Gudrun Göhring, MD, Michaela Scherr, PhD, Matthias Gaestel, PhD, Matthias Eder, MD, Julia Skokowa, MDPhD, Jianfeng Zhou, PhD, Karl Welte, Li-Gen Liu, MD, Arnold Ganser, MD, Zhixiong Li, MD

Blood (2018) 132 (Supplement 1): 771.

https://doi.org/10.1182/blood-2018-99-110770

Published: 2018

Journal Articles

TALEN ® -Based Gene Editing in Hematopoietic Stem Cells

Brian W Busser, PhD, Sonal Temburni, Alex Boyne, Alexandre Juillerat, Laurent Poirot, PhD, Philippe Duchateau

Blood (2018) 132 (Supplement 1): 5796.

https://doi.org/10.1182/blood-2018-99-117211

Published: 2018

Journal Articles

Engagement and Education of the Sickle Cell Disease Community: What Sickle Cell Disease Patients and Parents Want to Know about CRISPR Genome Editing

Stacy Desine, BA, Brittany Hollister, PhD, Anitra Persaud, BA, Vence L. Bonham, J.D.

Blood (2018) 132 (Supplement 1): 5805.

https://doi.org/10.1182/blood-2018-99-118735

Published: 2018

Journal Articles

Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34 + Cells from Patients with Sickle Cell Disease

Naoya Uchida, MD, Linhong Li, Juan J Haro-Mora, PhD, Selami Demirci, PhD, Tina Nassehi, Jackson Gamer, Claire Drysdale, Morgan Yapundich, Cornell Allen, Suk See De Ravin, MD PhD, Harry L Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD

Blood (2018) 132 (Supplement 1): 2191.

https://doi.org/10.1182/blood-2018-99-111677

Published: 2018

Journal Articles

Leukemogenic Pathway of MLL-AF4 Fusion-Positive Acute Lymphoblastic Leukemia

Mariko Eguchi, MD, Minenori Eguchi-Ishimae, MD PhD, Eiichi Ishii, MD PhD

Blood (2017) 130 (Supplement 1): 1230.

https://doi.org/10.1182/blood.V130.Suppl_1.1230.1230

Published: 2017

Journal Articles

Platelet-Targeted Gene Therapy of Hemophilia a Using Hematopoietic Stem Cells Derived from Genome-Edited Induced Pluripotent Stem Cells

Dawei Wang, Guowei Zhang, Junjie Gu, Xiaohu Shao, Xiaohong Pan, Shuxian Yao, Ying Jin, Qizhen Shi, MD PhD, Zhu Chen, PhD, Sai-Juan Chen, PhD

Blood (2017) 130 (Supplement 1): 2063.

https://doi.org/10.1182/blood.V130.Suppl_1.2063.2063

Published: 2017

Journal Articles

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Valentina Poletti, PhD, Sabine Charrier, PhD, Samia Martin, Bernard Gjata, Alban Vignaud, PhD, Fang Zhang, PhD, Karen Buckland, PhD, Michael Rothe, PhD, Axel Schambach, MD PhD, Sung-Yun Pai, MD, David A. Williams, MD, H. Bobby Gaspar, MD PhD, Adrian J Thrasher, MD PhD, Fulvio Mavilio, PhD

Blood (2016) 128 (22): 4705.

https://doi.org/10.1182/blood.V128.22.4705.4705

Published: 2016

Journal Articles

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

Adrian Schwarzer, MD PhD, Steven R Talbot, PhD, Oliver Dittrich-Breiholz, PhD, Adrian J Thrasher, MD PhD, Bobby Gaspar, MD, Ute Modlich, Christopher Baum, MD, Axel Schambach, MD PhD, Michael Rothe, PhD

Blood (2016) 128 (22): 4710.

https://doi.org/10.1182/blood.V128.22.4710.4710

Published: 2016

Journal Articles

Gene Modification of Human Hematopoietic Stem Cells with Suicide Genes As a Safety Control

Laurel Christine Truscott, MD, Tzu Ting Chiou, PhD, Roy L. Kao, MD, Satiro N. De Oliveira, MD

Blood (2016) 128 (22): 5888.

https://doi.org/10.1182/blood.V128.22.5888.5888

Published: 2016

Journal Articles

Resistant T-Cell Acute Lymphoblastic Leukemias That Emerge after In Vivo Treatment with Dexamethasone Frequently Down-Regulate Glucocorticoid Receptor Protein Expression

Blood (2016) 128 (22): 753.

https://doi.org/10.1182/blood.V128.22.753.753

Published: 2016

Journal Articles

Gene Therapy Studies in Hemoglobinopathies: Successes and Challenges

Marina Cavazzana, MD PhD

Blood (2016) 128 (22): SCI-50.

https://doi.org/10.1182/blood.V128.22.SCI-50.SCI-50

Published: 2016

Journal Articles

Correction of Fanconi Anemia Mutation Using the Crispr/Cas9 System

Nozomu Kawashima, MD, Yusuke Okuno, MD PhD, Yuko Sekiya, MD, Xinan Wang, PhD, Atsushi Narita, MD, Sayoko Doisaki, MD PhD, Michi Kamei, MD PhD, Nobuhiro Nishio, MD PhD, Hideki Muramatsu, MD PhD, Asahito Hama, MD PhD, Yoshiyuki Takahashi, MD PhD, Seiji Kojima, MD PhD

Blood (2015) 126 (23): 3622.

https://doi.org/10.1182/blood.V126.23.3622.3622

Published: 2015

Journal Articles

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Uimook Choi, PhD, Narda Theobald, Throm E Robert, PhD, John Gray, PhD, David J. Rawlings, MD, Brian P. Sorrentino, MD, Harry Malech, Suk See De Ravin, MD

Blood (2015) 126 (23): 2036.

https://doi.org/10.1182/blood.V126.23.2036.2036

Published: 2015

Journal Articles

Thymic expression of a T-cell receptor targeting a tumor-associated antigen coexpressed in the thymus induces T-ALL

Yongzhi Cui, Masahiro Onozawa, Haven R. Garber, Leigh Samsel, Ziyao Wang, J. Philip McCoy, Sandra Burkett, Xiaolin Wu, Peter D. Aplan, Crystal L. Mackall

Blood (2015) 125 (19): 2958–2967.

https://doi.org/10.1182/blood-2014-10-609271

Published: 2015

Includes: Supplemental data

Journal Articles

Generation and Analysis of a Novel Mouse Model for Chronic Myelomonocytic Leukemia (CMML) with Acquired Expression of c-CBL Q367P

Yuichiro Nakata, Takeshi Ueda, MD PhD, Akiko Nagamachi, PhD, Linda Wolff, PhD, Ogawa Seishi, MD, Toshiya Inaba, MD, Hiroaki Honda, MD PhD

Blood (2014) 124 (21): 4602.

https://doi.org/10.1182/blood.V124.21.4602.4602

Published: 2014

Journal Articles

Identifying Novel Genes and Signaling Pathways That Predispose to Therapy Related MDS (t-MDS)

Shailaja Akunuru, PhD, Kimberly Seymore, Rupali Kumar, Kalpana Nattamai, Hartmut Geiger

Blood (2014) 124 (21): 1905.

https://doi.org/10.1182/blood.V124.21.1905.1905

Published: 2014

NARROW

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

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

Homeobox Transcription Factor Hhex Promotes Myeloid Leukemia in Cooperation with Mutant ASXL1

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

Synergy between FLT3-ITD and p53 Haploinsufficiency or Loss in the Development of Acute Myeloid Leukemia

TALEN ® -Based Gene Editing in Hematopoietic Stem Cells

Engagement and Education of the Sickle Cell Disease Community: What Sickle Cell Disease Patients and Parents Want to Know about CRISPR Genome Editing

Development of a Clinically Applicable Method for High-Efficiency Gene Correction of Plerixafor-Mobilized CD34 + Cells from Patients with Sickle Cell Disease

Leukemogenic Pathway of MLL-AF4 Fusion-Positive Acute Lymphoblastic Leukemia

Platelet-Targeted Gene Therapy of Hemophilia a Using Hematopoietic Stem Cells Derived from Genome-Edited Induced Pluripotent Stem Cells

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

Gene Modification of Human Hematopoietic Stem Cells with Suicide Genes As a Safety Control

Resistant T-Cell Acute Lymphoblastic Leukemias That Emerge after In Vivo Treatment with Dexamethasone Frequently Down-Regulate Glucocorticoid Receptor Protein Expression

Gene Therapy Studies in Hemoglobinopathies: Successes and Challenges

Correction of Fanconi Anemia Mutation Using the Crispr/Cas9 System

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Thymic expression of a T-cell receptor targeting a tumor-associated antigen coexpressed in the thymus induces T-ALL

Generation and Analysis of a Novel Mouse Model for Chronic Myelomonocytic Leukemia (CMML) with Acquired Expression of c-CBL Q367P

Identifying Novel Genes and Signaling Pathways That Predispose to Therapy Related MDS (t-MDS)

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American Society of Hematology

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American Society of Hematology

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