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Journal Articles

Impaired ribosome biogenesis checkpoint activation induces p53-dependent MCL-1 degradation and MYC-driven lymphoma death

Ana Domostegui, Suresh Peddigari, Carol A. Mercer, Flavia Iannizzotto, Marta L. Rodriguez, Marta Garcia-Cajide, Virginia Amador, Sarah T. Diepstraten, Gemma L. Kelly, Ramón Salazar, Sara C. Kozma, Eric P. Kusnadi, Jian Kang, Antonio Gentilella, Richard B. Pearson, George Thomas, Joffrey Pelletier

Blood (2021) 137 (24): 3351–3364.

https://doi.org/10.1182/blood.2020007452

Published: 2021

Includes: Supplemental data

Journal Articles

Diamond-Blackfan anemia

Lydie Da Costa, Thierry Leblanc, Narla Mohandas

Blood (2020) 136 (11): 1262–1273.

https://doi.org/10.1182/blood.2019000947

Published: 2020

Journal Articles

Decoding ribosomes to keep growing (in AML)

Jungeun An, Myunggon Ko

Blood (2020) 135 (23): 2015–2016.

https://doi.org/10.1182/blood.2020006021

Published: 2020

Journal Articles

Distinct Evolutionary Patterns in Chronic Lymphocytic Leukemia (CLL) during Resistance to Graft-Versus-Leukemia (GvL)

Blood (2019) 134 (Supplement_1): 516.

https://doi.org/10.1182/blood-2019-129520

Published: 2019

Journal Articles

Pharmacological Inhibition of Nemo-like Kinase Rescues mTOR-Mediated Translation and Primes Progenitors for Leucine-Stimulated Erythroid Expansion in Pre-Clinical Models of Diamond Blackfan Anemia

Mark C Wilkes, PhD, Jacqueline D Mercado, Mallika Saxena, Jun Chen, MD PhD, Kavitha Siva, PhD, Hee-Don Chae, PhD, Minyoung Youn, PhD, Ethan Patrick Wentworth, Kevin Jung, Ryan Sathianathen, Hiral Patel, Bertil Glader, MD PhD, Anupama Narla, MD, Claire E Repellin, PhD, Hanna T Gazda, MD PhD, Manuel Serrano, PhD, Johan Flygare, MD PhD, Kathleen M. Sakamoto, MD PhD

Blood (2019) 134 (Supplement_1): 455.

https://doi.org/10.1182/blood-2019-129570

Published: 2019

Journal Articles

A Phase 1 First-in-Human Study of the Anti-CD38 Dimeric Fusion Protein TAK-169 for the Treatment of Patients (pts) with Relapsed or Refractory Multiple Myeloma (RRMM) Who Are Proteasome Inhibitor (PI)- and Immunomodulatory Drug (IMiD)-Refractory, Including Pts Relapsed/Refractory (R/R) or Naïve to Daratumumab (dara)

Shaji K. Kumar, MD, Robert F. Cornell, MD MS, Ola Landgren, MD PhD, Sikander Ailawadhi, MD, Jack P Higgins, PhD, Erin K Willert, Roger Waltzman, MD MBA, Jianchang Lin, PhD, Yuhong Zhang, Anya R Lublinsky, Ajeeta B Dash, PhD, Michael Hanley, Divya Manoharan, Morgan Leichter, Sean Ottinger, Richard J Labotka, John Newcomb, Alexander Vorog, MD

Blood (2019) 134 (Supplement_1): 1867.

https://doi.org/10.1182/blood-2019-125629

Published: 2019

Journal Articles

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Yuchi Honaker, PhD, Karen Sommer, PhD, Noelle Dahl, Yufei Xiang, MD, PhD, Christina Lopez, PhD, Samuel Scharffenberger, Michelle Christian, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD

Blood (2019) 134 (Supplement_1): 2071.

https://doi.org/10.1182/blood-2019-131946

Published: 2019

Journal Articles

Machine Learning Unveils Proteotypic Mimicry in Genetically Defined SCN Variants

Sebastian Hesse, MD, Christoph Klein, MD PhD, Rappsilber Juri, Prof., Yoko Mizoguchi, Monika Linder, PhD, Piotr Grabowski, PhD, Zahra Alizadeh, PhD, Zahra Pourpak, MD PhD, Nima Rezaei, MD PhD, Sorin Iurian, MD PhD, Ekrem Unal, MD, Fabian Hauck, MD PhD, Ralf Zimmer, Prof., Gergely Csaba, PhD

Blood (2019) 134 (Supplement_1): 3580.

https://doi.org/10.1182/blood-2019-131136

Published: 2019

Journal Articles

Identification of Novel Therapeutic Targets in Atm-Deficient Lymphomas Using a Whole Genome CRISPR/CAS-9 Screen

Tatjana Stankovic, MD PhD, Nicholas Davies, PhD, Louise J Tee, Andrew D Beggs, Malcolm Taylor, PhD

Blood (2019) 134 (Supplement_1): 1504.

https://doi.org/10.1182/blood-2019-129974

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

MCL-1 Inhibitor-Induced Killing of Multiple Myeloma Is Modulated By the Relative Level of Expression of Pro-Survival Members of the BCL-2 Family and the Proto-Oncogene MYC

Andrew Spencer, DM, Tiffany Khong, BSc, PhD, Maoshan Chenn, PhD

Blood (2019) 134 (Supplement_1): 1825.

https://doi.org/10.1182/blood-2019-127843

Published: 2019

Journal Articles

Casein Kinase 1 Delta Is a Novel Regulator of mRNA Translation and Druggable Target in Aggressive Lymphomas

Ipsita Pal, PhD, Sohani Das Sharma, PhD, Andre M. Grilo, PhD, Prabhjot S. Mundi, MD, Luigi Scotto, PhD, Abdullah Mahmood Ali, PhD, Luke E Berchowitz, PhD, Siddhartha Mukherjee, MD PhD, Owen O'Connor, MD PhD, Marko Jovanovic, PhD, Changchun Deng, MD PhD

Blood (2019) 134 (Supplement_1): 2864.

https://doi.org/10.1182/blood-2019-129528

Published: 2019

Journal Articles

Mutation of Murine Rpl5 reveals a New Model for Diamond Blackfan Anemia Characterized By Defective Erythropoiesis

Philippe Lemay, PhD, Morgan A Jones, MD PhD BS, MS, Fatma Mohamed, Marie-Claude Guyot, Alexander Ludlow, Qing Li, MD, James Douglas Engel, PhD, Renny Franceschi, PhD, Thomas L. Rothstein, MD PhD, Vesa Kaartinen, PhD, Zoha Kibar, PhD, Sharon A Singh, MD

Blood (2019) 134 (Supplement_1): 2495.

https://doi.org/10.1182/blood-2019-131526

Published: 2019

Journal Articles

Disease Modeling and Phenotype Rescue Using Inducible Pluripotent Stem Cells from Patients with Diamond-Blackfan Anemia

Senthil Velan Bhoopalan, MD PhD, Min-Joon Han, PhD, Steven Ellis, PhD, Harry Lesmana, MD, Jeremie H. Estepp, MD, Yoonjeong Jang, DVM,PhD, Mitchell J. Weiss, MD PhD, Marcin W. Wlodarski, MD PhD

Blood (2019) 134 (Supplement_1): 2496.

https://doi.org/10.1182/blood-2019-131354

Published: 2019

Journal Articles

MetAP2 Inhibition Modifies Hemoglobin S (HbS) to Delay Polymerization and Improve Blood Flow in Sickle Cell Disease

Melanie Demers, PhD, Sarah Sturtevant, BSc, Kevin Guertin, PhD, Dipti Gupta, Kunal Desai, MS, Benjamin Vieira, BS, Alexandra Hicks, PhD, Ayman Ismail, PhD, Yukio Nakamura, MD PhD, Bronner Goncalves, MD PhD, Giuseppe Di Caprio, PhD, Ethan Schonbrun, PhD, Scott Hansen, BS, Martin K. Safo, PhD, David K. Wood, PhD, John M. Higgins, MD PhD, David R. Light, PhD

Blood (2019) 134 (Supplement_1): 2260.

https://doi.org/10.1182/blood-2019-128727

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

Blood (2019) 134 (Supplement_1): 2262.

https://doi.org/10.1182/blood-2019-132078

Published: 2019

Journal Articles

Characterization of Congenital Sideroblastic Anemia Model Due to ABCB7 Defects: How Do Defects in Iron-Sulfur Cluster Metabolism Lead to Ring Sideroblast Formation?

Tohru Fujiwara, MD PhD, Chie Suzuki, Tetsuro Ochi, Koya Ono, MD, Kei Saito, Noriko Fukuhara, MD PhD, Yasushi Onishi, MD PhD, Hisayuki Yokoyama, MD PhD, Shinichi Fujimaki, Yukio Nakamura, MD PhD, Hideo Harigae, MD PhD

Blood (2019) 134 (Supplement_1): 2232.

https://doi.org/10.1182/blood-2019-123918

Published: 2019

Journal Articles

Whole Genome Sequencing Identifies Small Deletions in Ribosomal Genes Causing Diamond Blackfan Anemia

David M. Bodine, PhD, Jens Lichtenberg, Nancy Seidel, NISC Comparative SequencingProgram, G. Jayashree Jagadeesh, John G Conboy, PhD, Jason E. Farrar, MD, Jessica Kang, BS, Lionel Blanc, PhD, Evangelia Atsidaftos, MA, MPH, Steven Ellis, PhD, Jeffrey Michael M Lipton, MD PhD, Adrianna Vlachos, MD

Blood (2019) 134 (Supplement_1): 2502.

https://doi.org/10.1182/blood-2019-121710

Published: 2019

Journal Articles

The Ribosomal Biogenesis Inhibitor CX-5461 Is an Anti-Cancer Therapeutic That Increases Platelet Count in Mice and in Humans

Amandeep Kaur, PhD, Katherine M Hannan, PhD, Samina Nazir, MSc, Kylee H Maclachlan, FRACP,FRCPA, Gretchen Poortinga, PhD, Si Ming Man, PhD, Simon Harrison, MBBS, MRCP, FRCPath, FRACP, PhD, Ross D Hannan, PhD, Elizabeth E. Gardiner, PhD

Blood (2019) 134 (Supplement_1): 2360.

https://doi.org/10.1182/blood-2019-121713

Published: 2019

Journal Articles

Deconstructing the Clonal Advantage and Clonal Stability of 5q- Candidate Genes in Del(5q) MDS on a Single Cell Level

Ursula S.A. Stalmann, MD, Fabio Ticconi, MSc, Ronghui Li, MSc, Aaron B. Wong, PhD, Glenn Cowley, PhD, David E. Root, PhD, Dirk Heckl, PhD, Inge Snoeren, MSc, Sergio Martinez, PhDMSc, Tim H Brümmendorf, MD, Andreas Schuppert, PhD, Ivan Costa, PhD, Benjamin L. Ebert, MD PhD, Rebekka K. Schneider, MD PhD

Blood (2019) 134 (Supplement_1): 559.

https://doi.org/10.1182/blood-2019-122588

Published: 2019

NARROW

Impaired ribosome biogenesis checkpoint activation induces p53-dependent MCL-1 degradation and MYC-driven lymphoma death

Diamond-Blackfan anemia

Decoding ribosomes to keep growing (in AML)

Distinct Evolutionary Patterns in Chronic Lymphocytic Leukemia (CLL) during Resistance to Graft-Versus-Leukemia (GvL)

Pharmacological Inhibition of Nemo-like Kinase Rescues mTOR-Mediated Translation and Primes Progenitors for Leucine-Stimulated Erythroid Expansion in Pre-Clinical Models of Diamond Blackfan Anemia

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Machine Learning Unveils Proteotypic Mimicry in Genetically Defined SCN Variants

Identification of Novel Therapeutic Targets in Atm-Deficient Lymphomas Using a Whole Genome CRISPR/CAS-9 Screen

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

MCL-1 Inhibitor-Induced Killing of Multiple Myeloma Is Modulated By the Relative Level of Expression of Pro-Survival Members of the BCL-2 Family and the Proto-Oncogene MYC

Casein Kinase 1 Delta Is a Novel Regulator of mRNA Translation and Druggable Target in Aggressive Lymphomas

Mutation of Murine Rpl5 reveals a New Model for Diamond Blackfan Anemia Characterized By Defective Erythropoiesis

Disease Modeling and Phenotype Rescue Using Inducible Pluripotent Stem Cells from Patients with Diamond-Blackfan Anemia

MetAP2 Inhibition Modifies Hemoglobin S (HbS) to Delay Polymerization and Improve Blood Flow in Sickle Cell Disease

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

Characterization of Congenital Sideroblastic Anemia Model Due to ABCB7 Defects: How Do Defects in Iron-Sulfur Cluster Metabolism Lead to Ring Sideroblast Formation?

Whole Genome Sequencing Identifies Small Deletions in Ribosomal Genes Causing Diamond Blackfan Anemia

The Ribosomal Biogenesis Inhibitor CX-5461 Is an Anti-Cancer Therapeutic That Increases Platelet Count in Mice and in Humans

Deconstructing the Clonal Advantage and Clonal Stability of 5q- Candidate Genes in Del(5q) MDS on a Single Cell Level

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American Society of Hematology

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American Society of Hematology

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