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Journal Articles

GPS2 promotes erythroid differentiation by control of the stability of EKLF protein

Wen-Bing Ma, Xiao-Han Wang, Chang-Yan Li, Huan-Huan Tian, Jie Zhang, Jun-Jie Bi, Guang-Ming Ren, Shou-Song Tao, Xian Liu, Wen Zhang, Dong-Xu Li, Hui Chen, Yi-Qun Zhan, Miao Yu, Chang-Hui Ge, Xiao-Ming Yang, Rong-Hua Yin

Blood (2020) 135 (25): 2302–2315.

https://doi.org/10.1182/blood.2019003867

Published: 2020

Includes: Supplemental data

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Global Rnaseq/Proteomic-Phoshoproteomic Analysis Unveil Mir-21 As a Central Player in Driving Th17 Mediated Bone Disease in MM

Marco Rossi, MD, Emanuela Altomare, PhD, Cirino Botta, MD, Maria Eugenia Gallo Cantafio, Marco Gaspari, Domenico Taverna, Sarvide Sarai, Daniele Caracciolo, MD, Paola Critelli, MD, Francesco Conforti, Maria Teresa Di Martino, PhD, Bruno Paiva, PhD, Pierosandro Tagliaferri, MD, Pierfrancesco Tassone, MD

Blood (2019) 134 (Supplement_1): 505.

https://doi.org/10.1182/blood-2019-122429

Published: 2019

Journal Articles

The Transmembrane Receptor Roundabout 1 (ROBO1) Is Necessary for Multiple Myeloma Proliferation and Homing to the Bone Marrow Niche

Giada Bianchi, MD, Peter G. Czarnecki, MD, Matthew Ho, PhD, Aldo M. Roccaro, MD PhD, Antonio Sacco, RN, Yawara Kawano, MD PhD, Anil Aktas Samur, PhD, Annamaria Gulla, MD, Tianzeng Chen, Kenneth Wen, Xinchen Wu, Yu-Tzu Tai, PhD, Gulden Camci-Unal, PhD, Irene M. Ghobrial, MD, Ruben D. Carrasco, MD PhD, Kenneth C. Anderson, MD

Blood (2019) 134 (Supplement_1): 507.

https://doi.org/10.1182/blood-2019-128778

Published: 2019

Journal Articles

Integrated Epigenetic Profiling Identifies EZH2 As a Therapeutic Target to Re-Establish Immune Recognition of Leukemia Relapses with Loss of HLA Class II Expression

Valentina Gambacorta, MSc, Daniela Gnani, PhD, Laura Zito, MSc, Stefano Beretta, PhD, Lucia Zanotti, PhD, Oliveira Giacomo, PhD, Davide Cittaro, PhD, Ivan Merelli, PhD, Fabio Ciceri, MD, Raffaella Di Micco, PhD, Luca Vago, MD PhD

Blood (2019) 134 (Supplement_1): 514.

https://doi.org/10.1182/blood-2019-127395

Published: 2019

Journal Articles

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

Published: 2019

Journal Articles

Phase 1, Open-Label, Dose Escalation Study of I-131-CLR1404 (CLR 131) in Patients with Relapsed or Refractory Multiple Myeloma

Jarrod Longcor, Kate Oliver, BS

Blood (2019) 134 (Supplement_1): 1864.

https://doi.org/10.1182/blood-2019-131806

Published: 2019

Journal Articles

Gck Kinase Activity Is Critical for RAS Mutated Myeloma - a Potential Treatment Approach for Targeting Specific Mutations

Shirong Li, PhD, Jing Fu, PhD, Xiaoming Xu, PhD, Shixian Deng, PhD, Markus Y Mapara, MD PhD, Christophe Marcireau, Suzanne Lentzsch, MD PhD

Blood (2019) 134 (Supplement_1): 1813.

https://doi.org/10.1182/blood-2019-129531

Published: 2019

Journal Articles

Adoptive Therapy with Cord Blood Regulatory T Cells Can Treat Graft Vs Host Disease

Hongbing Ma, MD PhD, Ke Zeng, MD PhD, Mitsutaka Nishimoto, MD PhD, Mi-Ae Lyu, PhD, Meixian Huang, Swaminathan P. Iyer, MD, Simrit Parmar, MBBS

Blood (2019) 134 (Supplement_1): 1940.

https://doi.org/10.1182/blood-2019-129395

Published: 2019

Journal Articles

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Blood (2019) 134 (Supplement_1): 2058.

https://doi.org/10.1182/blood-2019-126746

Published: 2019

Journal Articles

The MEK Inhibitor Trametinib Enhances Diverse T Cell Reconstitution with Suppressing Xenogeneic Graft-Versus-Host-Disease

Hidekazu Itamura, MD PhD, Hiroyuki Muranushi, MD, Takero Shindo, MD PhD, Kazutaka Kitaura, DVM, PhD, Seiji Okada, MD PhD, Tadasu Shin-I, PhD, Ryuji Suzuki, DVM, PhD, Akifumi Takaori-Kondo, MD PhD, Shinya Kimura, MD PhD

Blood (2019) 134 (Supplement_1): 1929.

https://doi.org/10.1182/blood-2019-128913

Published: 2019

Journal Articles

A Novel Platelet-Targeting Fibrinolytic Strategy: Endocytosed Urokinase By Megakaryocytes Is Stored in α-Granules and Is Functional In Vivo in a Murine Model of Thrombosis

Hyunsook Ahn, MSc, John Tkaczynski, BS, Khalil Bdeir, PhD, Victoria Stepanova, Douglas B. Cines, MD, Mortimer Poncz, MD, Sergei Zaitsev

Blood (2019) 134 (Supplement_1): 3627.

https://doi.org/10.1182/blood-2019-124501

Published: 2019

Journal Articles

Utility of Newborn Screening for Severe Combined Immunodeficiency and X-Linked Agammaglobulinemia Using TREC and KREC Assays

Manabu Wakamatsu, MD, Hideki Muramatsu, MD PhD, Shinsuke Kataoka, MD, Yusuke Okuno, MD PhD, Sakai Yoshimi, Yoko Nakajima, MD PhD, Tetsuya Ito, MD PhD, Ikuya Tsuge, MD PhD, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 3604.

https://doi.org/10.1182/blood-2019-126669

Published: 2019

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

https://doi.org/10.1182/blood-2019-125743

Published: 2019

Journal Articles

Checkpoint Blockade in Combination with CD33 Chimeric Antigen Receptor T Cell Therapy and Hypomethylating Agent Against Acute Myeloid Leukemia

Tongyuan Xue, MD, Marissa Del Real, PhD, Emanuela Marcucci, Candida Toribio, Sonia Maryam Setayesh, Stephen J Forman, MD, David A Horne, PhD, Lihua E Budde, MD PhD

Blood (2019) 134 (Supplement_1): 1383.

https://doi.org/10.1182/blood-2019-121486

Published: 2019

Journal Articles

A Phase 2a Open-Label Study to Investigate Safety and Tolerability (including the MTD), Efficacy, Pharmacokinetics, Pharmacodynamics and Immunogenicity of MT-3724 in Combination with Lenalidomide in Subjects with Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma

Christine Burnett, Gary Gordon, MD PhD, Thomas Strack, MD, Tara Lehner, MS, PMP, Jack P Higgins, PhD, Jason Tache, DO

Blood (2019) 134 (Supplement_1): 1597.

https://doi.org/10.1182/blood-2019-128503

Published: 2019

Journal Articles

The Membrane Scaffold CD82 Regulates Acute Myeloid Leukemia Dormancy and Bone Marrow Niche Interactions

Muskan Floren, BS, Erica Pascetti, BS, Jennifer Gillette, PhD

Blood (2019) 134 (Supplement_1): 1396.

https://doi.org/10.1182/blood-2019-129320

Published: 2019

Journal Articles

Night Transplantation and Catecholamine Exposure Accelerates In Vivo Leukemia Development By Enhancing Bone Marrow Homing Capacity in Leukemic Cells

Pauline Hanns, MSc, Anna M Paczulla, PhD, Martina Konantz, PhD, Marcelle Baer, MSc, Pontus Lundberg, PhD, Stefan Dirnhofer, MD, Claudia Lengerke, MD

Blood (2019) 134 (Supplement_1): 1446.

https://doi.org/10.1182/blood-2019-126984

Published: 2019

Journal Articles

Effect of Rap-536 on MDS PDX-Murine Models with or without SF3B1 Mutation

Mathieu Meunier, Camille Knosp, Chloe Friedrich, Johanna Zannoni, Jean-Yves Cahn, MD PhD, Olivier Kosmider, PharmD, PhD, Michaela Fontenay, MD PhD, Sophie Park, MD PhD

Blood (2019) 134 (Supplement_1): 1705.

https://doi.org/10.1182/blood-2019-128407

Published: 2019

Journal Articles

A Novel Class of Bifunctional Immunotherapeutic That Exploits a Universal Antibody Binding Terminus (uABT) to Recruit Endogenous Antibodies to Cell Expressing CD38 Demonstrate In Vivo efficacy in Three Distinct Animal Models

Anna Bunin, PhD, Katy McGrath, BS, Ann Marie Rossi, MS, Matthew Welsch, PhD, Christian Vidal, PhD, David Trinh, BA, David Spiegel, MD, PhD, Luca Rastelli, PhD, Enrique Alvarez, DVM

Blood (2019) 134 (Supplement_1): 1820.

https://doi.org/10.1182/blood-2019-130838

Published: 2019

NARROW

GPS2 promotes erythroid differentiation by control of the stability of EKLF protein

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Global Rnaseq/Proteomic-Phoshoproteomic Analysis Unveil Mir-21 As a Central Player in Driving Th17 Mediated Bone Disease in MM

The Transmembrane Receptor Roundabout 1 (ROBO1) Is Necessary for Multiple Myeloma Proliferation and Homing to the Bone Marrow Niche

Integrated Epigenetic Profiling Identifies EZH2 As a Therapeutic Target to Re-Establish Immune Recognition of Leukemia Relapses with Loss of HLA Class II Expression

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Phase 1, Open-Label, Dose Escalation Study of I-131-CLR1404 (CLR 131) in Patients with Relapsed or Refractory Multiple Myeloma

Gck Kinase Activity Is Critical for RAS Mutated Myeloma - a Potential Treatment Approach for Targeting Specific Mutations

Adoptive Therapy with Cord Blood Regulatory T Cells Can Treat Graft Vs Host Disease

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

The MEK Inhibitor Trametinib Enhances Diverse T Cell Reconstitution with Suppressing Xenogeneic Graft-Versus-Host-Disease

A Novel Platelet-Targeting Fibrinolytic Strategy: Endocytosed Urokinase By Megakaryocytes Is Stored in α-Granules and Is Functional In Vivo in a Murine Model of Thrombosis

Utility of Newborn Screening for Severe Combined Immunodeficiency and X-Linked Agammaglobulinemia Using TREC and KREC Assays

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Checkpoint Blockade in Combination with CD33 Chimeric Antigen Receptor T Cell Therapy and Hypomethylating Agent Against Acute Myeloid Leukemia

A Phase 2a Open-Label Study to Investigate Safety and Tolerability (including the MTD), Efficacy, Pharmacokinetics, Pharmacodynamics and Immunogenicity of MT-3724 in Combination with Lenalidomide in Subjects with Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma

The Membrane Scaffold CD82 Regulates Acute Myeloid Leukemia Dormancy and Bone Marrow Niche Interactions

Night Transplantation and Catecholamine Exposure Accelerates In Vivo Leukemia Development By Enhancing Bone Marrow Homing Capacity in Leukemic Cells

Effect of Rap-536 on MDS PDX-Murine Models with or without SF3B1 Mutation

A Novel Class of Bifunctional Immunotherapeutic That Exploits a Universal Antibody Binding Terminus (uABT) to Recruit Endogenous Antibodies to Cell Expressing CD38 Demonstrate In Vivo efficacy in Three Distinct Animal Models

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American Society of Hematology

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American Society of Hematology

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