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Journal Articles

PRMT5 Inhibition Modulates E2F1 Methylation and Gene Regulatory Networks Leading to Therapeutic Efficacy in JAK2VF Mutant MPN

Friederike Pastore, MD, Neha Bhagwat, PhDBS, Aishwarya Krishnan, Alessandro Pastore, Bing Li, MD, Robert L Bowman, PhD, Wenbin Xiao, MD PhD, Aaron D Viny, MDMS, Abdul Karzai, Anouar Zouak, Young C Park, Keith Cordner, Stephanie Braunstein, Alexander Grego, Jaanvi Mehta, Benjamin H Durham, MD, Richard P. Koche, PhD, Jesper Maag, Peggy Scherle, PhD, Kris Vaddi, PhD, Ross L. Levine, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 473.
https://doi.org/10.1182/blood-2019-127122
Published: 2019
Journal Articles

A Novel Non-Covalent Modulator of Hemoglobin Improves Anemia and Reduces Sickling in a Mouse Model of Sickle Cell Disease

Kelly M. Knee, PhD, Reema Jasuja, PhD, Amey Barakat, MS, Dharani Rao, Zane Wenzel, MS, Parag Sahasrabudhe, Jatin Narula, PhD, Jay Jasti, Jeanne S. Chang, Kevin Beaumont, David W. Piotrowski, Jay M. Janz, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 207.
https://doi.org/10.1182/blood-2019-128270
Published: 2019
Journal Articles

Formin Links Erythroid Cytoskeleton to Organelle Clearance through Escrt-III Complex during Reticulocyte Maturation

Yijie Liu, PhD, Haiyan Bao, MD, Yang Mei, PhD, Xu Han, PhD, Peng Ji, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 151.
https://doi.org/10.1182/blood-2019-125157
Published: 2019
Journal Articles

VPS4A mutations Cause a Syndrome with Dyserythropoiesis, Hemolytic Anemia, and Neurodevelopmental Delay

Katie Giger Seu, PhD, Lisa Trump, PhD, Sana Emberesh, MBBS, Robert B. Lorsbach, MD PhD, Clarissa E. Johnson, MD, Jessica Meznarich, MD, Hunter Underhill, MD PhD, Haripriya Sakthivel, Omar Niss, MD, Shannon Nortman, Lionel Blanc, PhD, Wenying Zhang, MD PhD, Carolyn Lutzko, PhD, Theodosia A. Kalfa, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 339.
https://doi.org/10.1182/blood-2019-128230
Published: 2019
Journal Articles

Complete Spatial Mapping of Steady-State Erythropoiesis

Qingqing Wu, PhD, Jizhou Zhang, Courtney Johnson, BS, Anastasiya Slaughter, BA, Margot Lindsay May, BS, Daniel Lucas-Alcaraz, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 274.
https://doi.org/10.1182/blood-2019-129122
Published: 2019
Journal Articles

A Case of Mistaken Identity: TTP-like State Due to Heroin Overdose

Phani Borra, MD, Cory Edward Mellon, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4905.
https://doi.org/10.1182/blood-2019-128537
Published: 2019
Journal Articles

Caplacizumab As Step-up Therapy in Refractory TTP Utilizing Adamts-13 Activity and Evidence of End-Organ Damage

Neel Belani, MD, Adriel Barrios-Anderson, Sc.B, A.B., William Rafelson, MDMBA, Matthew I. Quesenberry, MD, Rabin Niroula, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 4909.
https://doi.org/10.1182/blood-2019-123206
Published: 2019
Journal Articles

Beyond the Routine CBC: Research CBC Parameters Associated with Myelodysplastic Syndromes and Underlying Mutations

Olga Pozdnyakova, MD PhD, Radu Stefan Niculescu, PhD, Tracey Kroll, BSc, Lisa Golemme, MLS (ASCP), MS, Nolan Raymond, MLS (ASCP), Debra Briggs, MT(ASCP), MS, Annette S. Kim, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5419.
https://doi.org/10.1182/blood-2019-125654
Published: 2019
Journal Articles

Ex vivo Engineering of Red Blood Cells

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

Correlation of Voxelotor Exposure with Hemoglobin Response and Measures of Hemolysis in Patients from the HOPE Study

Jo Howard, MB, BChir, Elliott Vichinsky, MD UCSF, Jennifer Knight-Madden, MBBS, PhD, Margaret Tonda, PharmD, Carla Washington, Barbara Tong, PhD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Victor R Gordeuk, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1020.
https://doi.org/10.1182/blood-2019-130802
Published: 2019
Journal Articles

Effects of Targeting the Interleukin-1 Receptor in a Murine Model of Sickle Cell Disease

Jessica Venugopal, PhD, Jintao Wang, PhD, Chiao Guo, Daniel Eitzman, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2259.
https://doi.org/10.1182/blood-2019-128980
Published: 2019
Journal Articles

Improvement in Red Blood Cell Physiology in Children with Sickle Cell Anemia Receiving Voxelotor

Satheesh Chonat, MD, Earl Fields, Hannah Baratz, Amanda Watt, Mira Pochron, PhD, Sandy Dixon, Margaret Tonda, PharmD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Clark Brown, MD PhD, David R. Archer, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2281.
https://doi.org/10.1182/blood-2019-129351
Published: 2019
Journal Articles

Hypoxia Dependent and Independent Dysregulation of the Transcriptome in Sickle Cell Anemia

Xu Zhang, PhD, Jihyun Song, PhD, Binal N. Shah, Taif Hassan, Galina Miasnikova, MD, Adelina Sergueeva, MD, Josef T. Prchal, MD, Victor R Gordeuk, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2262.
https://doi.org/10.1182/blood-2019-132078
Published: 2019
Journal Articles

High Natural Killer Cell Count at Diagnosis Predicts a Good Response to Immunosuppressive Therapy in Aplastic Anemia

Atsushi Narita, Hideki Muramatsu, MD PhD, Yinyan Xu, Motoharu Hamada, MD PhD, Daisuke Ichikawa, MD, Eri Nishikawa, MD PhD, Nozomu Kawashima, MD, Yusuke Okuno, MD PhD, Nobuhiro Nishio, MD PhD, Hirohito Yamazaki, MD PhD, Shinji Nakao, MD PhD, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2505.
https://doi.org/10.1182/blood-2019-124945
Published: 2019
Journal Articles

Establishment of a Spontaneous Stromal Microenvironment from Cord Blood Supports Human Dynamic Erythropoietic Temporal Maturation in Long-Term Serum- and Cytokine-Free 3D Cultures and Reveals a Distinct CD44 hi Population

Susana Brito Dos Santos, MSc PhD, Athanasios Mantalaris, PhD, Nicki Panoskaltsis, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2219.
https://doi.org/10.1182/blood-2019-131085
Published: 2019
Journal Articles

Antibodies Against the Erythroferrone N-Terminal Domain Prevent Hepcidin Suppression and Ameliorate Murine Thalassemia

Joao Arezes, BS, MSc, PhD, Niall Foy, Susan Benard, Anagha Sawant, May S Tam, Zachary Maben, Kirsty McHugh, Doris Quinkert, Andrew E Armitage, Sant-Rayn Pasricha, MD PhD, Pei-Jin Lim, Joe Frost, Edward LaVallie, Orla Cunningham, PhD, Matthew Lambert, Debra D Pittman, MS, John E. Murphy, PhD, Simon J Draper, Reema Jasuja, PhD, Alexander Drakesmith
Journal: Blood
Blood (2019) 134 (Supplement_1): 964.
https://doi.org/10.1182/blood-2019-130246
Published: 2019
Journal Articles

A New Role of Glutathion Peroxydase 4 in Human Erythroblast Enucleation

Hakim Ouled-Haddou, Kahia Messaoudi, Roggiero Lopes Dos Santos, Candice Carola, MD, Yohann Demont, Alexis Caulier, Pascal Vong, Nicolas Jankovsky, Delphine Lebon, MD, Jessica Platon, Julien Demagny, Nicolas Guillaume, Jean-Pierre Marolleau, MD PhD, Jacques Rochette, MB, DSc, Loïc Garçon
Journal: Blood
Blood (2019) 134 (Supplement_1): 938.
https://doi.org/10.1182/blood-2019-122161
Published: 2019
Journal Articles

Phase I Study of Cord Blood Transplantation with Intra-Bone Marrow Injection of Mesenchymal Stem Cells

Tatsunori Goto, MD, Makoto Murata, MD PhD, Tetsuya Nishida, MD PhD, Seitaro Terakura, MD PhD, Sonoko Kamoshita, MD, Yuichi Ishikawa, MD PhD, Yoko Ushijima, MD PhD, Yoshiya Adachi, MD PhD, Katsuyoshi Kato, PhD, Akihiro Hirakawa, PhD, Satoshi Nishiwaki, MD PhD, Nobuhiro Nishio, MD PhD, Yoshiyuki Takahashi, MD PhD, Yoshihisa Kodera, MD, Tadashi Matsushita, Hitoshi Kiyoi, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2004.
https://doi.org/10.1182/blood-2019-122101
Published: 2019
Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2066.
https://doi.org/10.1182/blood-2019-131318
Published: 2019
Journal Articles

Proof of Concept for Repair of Sickle Cell Disease By Non-Myeloablative MHC Disparate T Cell Depleted HSCT Combined with Donor-Derived Veto Cells

Aloukick Kumar Singh, PhD, Elias Schetzen, PhD, Esther BACHAR Lustig, PhD, Wei-Hsin Liu, M.Sc, Raj Kumar Yadav, PhD, Sandeep Kumar Yadav, PhD, Kathryn McGinnis, M.Sc, Yair Reisner, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1919.
https://doi.org/10.1182/blood-2019-124594
Published: 2019