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Journal Articles

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

Free
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2242.
https://doi.org/10.1182/blood-2019-124043
Published: 2019
Journal Articles

Myeloma Derived Extracellular Vesicles Containing Endoplasmic Reticulum Remodel the Bone Marrow Microenvironment Towards a Pro-Tumoral Senescent Phenotype

Free
Yu Sun, BSc, MSc, PhD, Jayna J Mistry, BSc, Jamie A Moore, BSc, MSc, Charlotte Hellmich, MBBChir, Christopher R Marlein, PhD, Anna Haestier, Cesar A. Gomez, MD PhD FRCPath, Christopher J Morris, PhD, Kristian M Bowles, MBBS, PhD, Stuart A Rushworth, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 689.
https://doi.org/10.1182/blood-2019-128268
Published: 2019
Journal Articles

Heterozygous Mutations of Clpb As a Newly Identified and Frequent Cause of Severe Congenital Neutropenia

Free
Julia T Warren, MD PhD, Peeradol Wattanasirakul, David H Spencer, MD, Adam Locke, PhD, Vahagn Makaryan, MD, Audrey Anna Bolyard, RN, BS, Karl Welte, Julia Skokowa, MD PhD, David C. Dale, MD, Daniel C. Link, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 433.
https://doi.org/10.1182/blood-2019-129635
Published: 2019
Journal Articles

Interrogation of Individual CLL Loss-of-Function Lesions By CRISPR In Vivo Editing Reveals Common and Unique Pathway Alterations

Free
Elisa Ten Hacken, PhD, Shanye Yin, PhD, Kendell Clement, PhD, Robert A. Redd, MS, Maria Hernandez-Sanchez, PhD, Shuqiang Li, PhD, Michaela Gruber, MD PhD, Heather Joyal, Geoffrey Fell, MS, Shu Wang, Jose Jacob, James Flynn, David Ruff, Keith W. Jones, Lili Wang, MD PhD, Donna S Neuberg, ScD, Luca Pinello, PhD, Kenneth J. Livak, PhD, Ruben D. Carrasco, MD PhD, Catherine J. Wu, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 684.
https://doi.org/10.1182/blood-2019-127673
Published: 2019
Journal Articles

Targeting Transcriptional Co-Activator PGC-1a for the Treatment of Sickle Cell Disease

Free
Alawi Habara, MBBS, MSc, Cuong LE, PhD, David H.K. Chui, MD, Martin H. Steinberg, MD, Shuaiying Cui, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2254.
https://doi.org/10.1182/blood-2019-123959
Published: 2019
Journal Articles

Ex vivo Engineering of Red Blood Cells

Free
Jan Frayne, BsC, PhD, Deborah E Daniels, Jan Frayne, BsC PhD, Timothy J Satchwell, PhD, Joseph Hawksworth, BSc PhD, Belinda K Singleton, Tatyana N Andrienko, PhD, Marieangela C Wilson, Sabine Kupzig, Marjolein Meinders, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Ivan Ferrer Vicens, PhD MSc, BSc, Rebecca Griffiths, PhD, Nicola Cogan, Kongtana Trakarnsanga, MD PhD, David J. Anstee, PhD FRCPath, Ashley Mark Toye, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-5.
https://doi.org/10.1182/blood-2019-121117
Published: 2019
Journal Articles

Engineering High Affinity and Cleavage Resistant CD16 to Augment ADCC of Placental Hematopoietic Stem Cells-Derived Natural Killer Cells

Free
Xuan Guo, PhD, Srinivas Somanchi, PhD, Rohit Mathur, PhD, Shuyang He, PhD, Qian Ye, PhD, Andrea Difiglia, BS, Salvatore Rotondo, MS, Hemlata Rana, MS, Weifang Ling, PhD, James Edinger, PhD, Robert Hariri, MD PhD, Xiaokui Zhang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1576.
https://doi.org/10.1182/blood-2019-127009
Published: 2019
Journal Articles

Single Cell Clones Derived from a Patient's AML Xenograft Display Genetic and Functional Heterogeneity

Free
Christina Maria Zeller, MSc, Daniel Richter, MSc, Binje Vick, PhD, Tobias Herold, MD, Johannes Bagnoli, Maja Rothenberg-Thurley, PhD, Klaus H. Metzeler, MD, Karsten Spiekermann, MD, Wolfgang Enard, PhD, Irmela Jeremias, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1450.
https://doi.org/10.1182/blood-2019-127867
Published: 2019
Journal Articles

Acute Myeloid Leukemia Export Mitochondria in Extracellular Vesicles Which Induces Pro-Tumoral Changes in Bone Marrow Macrophages

Free
Jamie A Moore, BSc, MSc, Jayna J Mistry, BSc, Rebecca H Horton, Charlotte Hellmich, MBBChir, Adam Pattinson, MSc, BSc, Adam Morfakis, BSc, Yu Sun, BSc, MSc, PhD, Matthew Lawes, MD, Christopher J Morris, PhD, Anna Haestier, Kristian M Bowles, MBBS, PhD, Stuart A Rushworth, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1427.
https://doi.org/10.1182/blood-2019-128447
Published: 2019
Journal Articles

CBFA2T3-GLIS2 induces Malignant Transformation of Primitive CD34+ Cord Blood Cells and Recapitulates Infant Megakaryocytic Leukemia

Free
Tiffany A. Hylkema, BS, Laura Pardo, Keith R. Loeb, MD, Quy Le, PhD, Suzan Imren, MD, Lisa Eidenschink Brodersen, PhD, Michael R. Loken, PhD, Soheil Meshinchi, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1444.
https://doi.org/10.1182/blood-2019-126185
Published: 2019
Journal Articles

Leukemia Burden in Peripheral Blood Has Negative Impact on CD19-CART Cell Culture and Its Clinical Efficacy in Patients with Refractory or Relapsed B Cell Acute Lymphoblastic Leukemia

Free
Biping Deng, BS, MD, Jing Pan, Shuangyou Liu, Yuehui Lin, Zhaoli Liu, Yunlong Chen, Xiaoming Qu, Yu 'e Zhang, Zhongxin Zhang, Xuansha Niu, Ming Ma, Tong Wu, MD, Alex Hong Chang, Chunrong Tong
Journal: Blood
Blood (2019) 134 (Supplement_1): 1957.
https://doi.org/10.1182/blood-2019-126695
Published: 2019
Journal Articles

High Mobility Group A1 Chromatin Regulators Function As Critical Drivers of Leukemogenesis in Preclinical Models of Relapsed, Pediatric B-Cell ALL

Free
Liping Li, MD PhD, Katharina Hayer, Lingling Xian, MD PhD, Li Luo, PhD, Leslie Cope, Andrei Thomas-Tikhonenko, PhD, Linda Resar, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3946.
https://doi.org/10.1182/blood-2019-130261
Published: 2019
Journal Articles

Dead/H-Box Helicase 11 ( DDX11 ) Mutations Correlate with Increased Relapse Risk in Persons with Acute Myeloid Leukaemia and Promote Proliferation and Survival of Human AML Cells in Vitro and in Immune Deficient Mice

Free
Ya-Lan Zhou, MD, Li-Xin Wu, Robert Peter Gale, Zi-Long Wang, Jin-Lan Li, Hao Jiang, Qian Jiang, MD, Bin Jiang, Shan-Bo Cao, Ying Sun, Feng Lou, Chengcheng Wang, Yan-Rong Liu, Yu Wang, Ying-Jun Chang, Lanping Xu, Xiaohui Zhang, MD, Kaiyan Liu, Guorui Ruan, Xiaojun Huang, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2732.
https://doi.org/10.1182/blood-2019-127831
Published: 2019
Journal Articles

PRMT5 Is a Key Epigenetic Regulator That Promotes Transcriptional Activation in Mantle Cell Lymphoma By Regulating the Lysine Methyltransferase SETD7 and MLL1 Activity

Free
JI Hyun Chung, PhD, Shelby Sloan, BS, Peggy Scherle, PhD, Kris Vaddi, PhD, Said Sif, PhD, Rosa Lapalombella, PhD, Robert A Baiocchi, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2777.
https://doi.org/10.1182/blood-2019-131020
Published: 2019
Journal Articles

Clinical and Functional Studies Reveal That TP73 Isoforms Levels Are Associated with Prognosis and RA-Resistance in Acute Promyelocytic Leukemia

Free
César Alexander Ortiz Rojas, BSc, Diego A Pereira-Martins, MSc, Isabel Weinhäuser, MSc, Luciana Yamamoto Almeida, PhD, Thiago Mantello Bianco, MSc, Lorena Lobo Figueiredo-Pontes, MD PhD, Luisa C A Koury, MD, Raul Antônio Morais Melo, MD PhD, Rosane Bittencourt, Katia B Pagnano, MD PhD, Ricardo Pasquini, MD, Elenaide C. Nunes, MD, Evandro Maranhão Fagundes, MD, Ana Beatriz F. Gloria, MD, Fabio R. Kerbauy, MD, Armand Keating, MD, Martin S. Tallman, MD, Raul Ribeiro, MD, Richard Dillon, MA, PhD MRCP, FRCPath, Arnold Ganser, MD, Bob Löwenberg, MD PhD, Peter Valk, PhD, Miguel A. Sanz, Nancy Berliner, MD, Eduardo M Rego, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2719.
https://doi.org/10.1182/blood-2019-131211
Published: 2019
Journal Articles

Germline Mutations Predispose to Familial Myeloproliferative Neoplasms

Free
Stephanie Berg, DO, Manuel O. Diaz, MD, Sucha Nand, MD, Jiwang Zhang, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2969.
https://doi.org/10.1182/blood-2019-124805
Published: 2019
Journal Articles

CCL2 Promotes Macrophages-Associated Chemoresistance Via MCPIP1 Dual Catalytic Activities in Multiple Myeloma

Free
Ruyi Xu, Yi Li, MD, Haimeng Yan, MD, Xi Huang, MD, He Huang, MD PhD, Zhen Cai, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3086.
https://doi.org/10.1182/blood-2019-126498
Published: 2019
Journal Articles

Fto Promotes Extramedullary Progression of Multiple Myeloma By Regulation of HSF1 through m 6 a RNA Methylation

Free
Aoshuang Xu, MD, Jiasi Zhang, MD, Liping Zuo, MD, Jian Xu, PhD, Xuejiao Yin, MD, Qianwen Cheng, MD, Yuyang Zhang, MD, Lei Chen, PhD, Fei Zhao, PhD, Fengjuan Fan, PhD, Yu Hu, Chunyan Sun, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3063.
https://doi.org/10.1182/blood-2019-125635
Published: 2019
Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Free
Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 3350.
https://doi.org/10.1182/blood-2019-127352
Published: 2019
Journal Articles

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

Free
Stefan Radtke, PhD, Margaret Cui, Anai M Perez, Yan-Yi Chan, Stefanie Schmuck, BS, Andrew J Berger, PhD, Thomas Eunson, BSc, Hans-Peter Kiem, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3246.
https://doi.org/10.1182/blood-2019-125969
Published: 2019