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Journal Articles

Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

Blood (2021) 137 (18): 2450–2462.

https://doi.org/10.1182/blood.2020009620

Published: 2021

Includes: Supplemental data

Journal Articles

How I treat warm autoimmune hemolytic anemia

Wilma Barcellini, Bruno Fattizzo

Blood (2021) 137 (10): 1283–1294.

https://doi.org/10.1182/blood.2019003808

Published: 2021

Journal Articles

The neurotransmitter receptor Gabbr1 regulates proliferation and function of hematopoietic stem and progenitor cells

Lijian Shao, Adedamola Elujoba-Bridenstine, Katherine E. Zink, Laura M. Sanchez, Brian J. Cox, Karen E. Pollok, Anthony L. Sinn, Barbara J. Bailey, Emily C. Sims, Scott H. Cooper, Hal E. Broxmeyer, Kostandin V. Pajcini, Owen J. Tamplin

Blood (2021) 137 (6): 775–787.

https://doi.org/10.1182/blood.2019004415

Published: 2021

Includes: Supplemental data

Journal Articles

Germline TET2 loss of function causes childhood immunodeficiency and lymphoma

Blood (2020) 136 (9): 1055–1066.

https://doi.org/10.1182/blood.2020005844

Published: 2020

Includes: Supplemental data

Journal Articles

IL-7R is essential for leukemia-initiating cell activity of T-cell acute lymphoblastic leukemia

Sara González-García, Marta Mosquera, Patricia Fuentes, Tiziana Palumbo, Adela Escudero, Antonio Pérez-Martínez, Manuel Ramírez, Anne E. Corcoran, Maria L. Toribio

Blood (2019) 134 (24): 2171–2182.

https://doi.org/10.1182/blood.2019000982

Published: 2019

Includes: Supplemental data

Journal Articles

Retrospective and Prospective Study of Childhood Autoimmune Hemolytic Anemia. a Preliminary Report from the Red Cell Working Group of the Paediatric Hemato-Oncology Italian Associations (AIEOP)

Blood (2019) 134 (Supplement_1): 947.

https://doi.org/10.1182/blood-2019-124735

Published: 2019

Journal Articles

Common Variable Immunodeficiency (CVID) in Adults As First Manifestation of (cryptic) Dyskeratosis Congenita

Benjamin Rolles, MD, Alla Bulashevska, PhD, Michele Proietti, MD, Sigune Goldacker, MD, Klaus Warnatz, MD, Nadezhda Camacho-Ordonez, MD, Margherita Vieri, MSc, Martin Kirschner, MD, Tim H Brümmendorf, MD, Bodo Grimbacher, MD, Fabian Beier, MD

Blood (2019) 134 (Supplement_1): 1217.

https://doi.org/10.1182/blood-2019-128915

Published: 2019

Journal Articles

Hyper-Sialylated IgG M254, an Innovative Therapeutic Candidate, Evaluated in Healthy Volunteers and in Patients with Immune Thrombocytopenia Purpura: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics

Santiago Arroyo, MD PhD, Renger G Tiessen, MD, William S Denney, PhD, Jim Jin, MS, Mattheus Paulus (Thijs) van Iersel, MD, Heidi Zeitz, MD CCRA, Anthony M Manning, PhD, Martin R. Schipperus, MD PhD, James B. Bussel, MD

Blood (2019) 134 (Supplement_1): 1090.

https://doi.org/10.1182/blood-2019-125993

Published: 2019

Journal Articles

GATA2 -Dependent Developmental and Regenerative Networks

Alexandra Soukup, PhD, Kirby D Johnson, PhD, Daniel J Conn, PhD, Evgenia Shishkova, PhD, Koichi Ricardo Katsumura, MD PhD, Peng Liu, PhD, Erik A. Ranheim, MD PhD, Katherine R. Calvo, MD PhD, Amy P. Hsu, Steven M. Holland, MD, Joshua J Coon, PhD, Sunduz Keles, PhD, Emery H Bresnick, PhD

Blood (2019) 134 (Supplement_1): 1182.

https://doi.org/10.1182/blood-2019-126875

Published: 2019

Journal Articles

Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34 + Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major

Riguo Fang, PhD, Pengfei Yuan, PhD, Lingling Yu, Bachelor, Huihui Yang, Master, Jie Liu, Master, Jia Shi, PhD, Yongjian Zhang, PhD, Wei Zhang, PhD, Yiding Zhao, PhD, Zhiyong Peng, MD, Chunfu Li, MD, Yun Li, MD, Dong Wei, PhD

Blood (2019) 134 (Supplement_1): 965.

https://doi.org/10.1182/blood-2019-126499

Published: 2019

Journal Articles

Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia

Shuquan Rao, PhD, Josias Brito-Frazao, Anna V. Serbin, Qiuming Yao, PhD, Kevin Luk, Yuxuan Wu, PhD, Jing Zeng, MD, Chunyan Ren, PhD, Ruth Watkinson, Myriam Armant, PhD, Luca Pinello, PhD, Akiko Shimamura, MD PhD, Benhur Lee, Scot A. Wolfe, PhD, Peter E. Newburger, MD, Daniel E. Bauer, MD PhD

Blood (2019) 134 (Supplement_1): 3.

https://doi.org/10.1182/blood-2019-131073

Published: 2019

Journal Articles

Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference

Naoya Uchida, MD, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Tina Nassehi, Matthew Wielgosz, Francesca Ferrara, PhD, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Byoung Y. Ryu, PhD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 2063.

https://doi.org/10.1182/blood-2019-122770

Published: 2019

Journal Articles

Potential Influence of Hematological or Immunological Hereditary Predisposition Genes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematological Malignancies

Zhihui Li, Qinlong Zheng, Yongqiang Zhao, Yanzhi Song, Fan Yang, Min Jiang, Chunrong Tong, Tong Wu, MD

Blood (2019) 134 (Supplement_1): 2037.

https://doi.org/10.1182/blood-2019-126339

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

Blood (2019) 134 (Supplement_1): 2066.

https://doi.org/10.1182/blood-2019-131318

Published: 2019

Journal Articles

Activation of the Receptor Tyrosine Kinase (RET) By GDNF/GFRα1 Improves Cord Blood-Derived HSC in Vitro expansion and In Vivo engraftment

William Grey, PhD MSc, Rakhee Chauhan, PhD, Manuel Garcia-Albornoz, Hector Huerga Encabo, Neil McDonald, PhD, Dominique Bonnet, PhD

Blood (2019) 134 (Supplement_1): 1924.

https://doi.org/10.1182/blood-2019-121331

Published: 2019

Journal Articles

iPSC-Derived NK Cells Synergize with T Cells and Anti-PD-1 Antibody to Mediate Durable Anti-Tumor Responses In Vivo

Jeffrey S. Miller, MD, Ryan Bjordahl, PhD, Svetlana Gaidarova, MS, Sajid Mahmood, PhD, Paul Rogers, PhD, Ge Moyar, Bruce R. Blazar, MD, Dan S Kaufman, MD PhD, Bahram Valamehr, PhD, Frank Cichocki, PhD

Blood (2019) 134 (Supplement_1): 1933.

https://doi.org/10.1182/blood-2019-124961

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

Prophylactic Itacitinib (INCB039110) for the Prevention of Cytokine Release Syndrome Induced By Chimeric Antigen Receptor T-Cells (CAR-T-cells) Therapy

Eduardo Huarte, PhD, Roddy S O'Connor, PhD, Melissa Parker, Taisheng Huang, Michael C. Milone, Paul Smith

Blood (2019) 134 (Supplement_1): 1934.

https://doi.org/10.1182/blood-2019-128288

Published: 2019

Journal Articles

Secondary Autoimmune Neutropenia: Data from the Italian Neutropenia Registry

Francesca Fioredda, PhD, Piero Farruggia, Andrea Beccaria, Antonino Trizzino, LuciaDora Notarangelo, Tiziana Lanza, Andrea Rotulo, Isabella Ceccherini, Angela Guarina, Alice Grossi, Federico Verzegnassi, Elena Mastrodicasa, Laura Luti, Angelica Barone, Giovanna Russo, Laura Porretti, Marta Pillon, Maurizio Miano, Carlo Dufour

Blood (2019) 134 (Supplement_1): 3585.

https://doi.org/10.1182/blood-2019-126526

Published: 2019

Journal Articles

Vaccine Response to a Neo Polyssacharide Antigen Typhim Vi on the Identification of Secondary Immunodeficiencies in Hematological Malignancy

Juliana Ochoa-Grullón, Celina Benavente Cuesta, G Cordero Torres, Cristina Pérez López, Ascensión Peña Cortijo, Antonia Rodríguez de la Peña, Ana Álvarez Carmona, Marta Mateo Morales, Edgard Rodríguez de Frías, Kissy Guevara-Hoyer, Miguel Fernández-Arquero, Lidia Fernández-Paredes, Rebeca Perez de Diego, Silvia Sánchez-Ramón

Blood (2019) 134 (Supplement_1): 3600.

https://doi.org/10.1182/blood-2019-129387

Published: 2019

NARROW

Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

How I treat warm autoimmune hemolytic anemia

The neurotransmitter receptor Gabbr1 regulates proliferation and function of hematopoietic stem and progenitor cells

Germline TET2 loss of function causes childhood immunodeficiency and lymphoma

IL-7R is essential for leukemia-initiating cell activity of T-cell acute lymphoblastic leukemia

Retrospective and Prospective Study of Childhood Autoimmune Hemolytic Anemia. a Preliminary Report from the Red Cell Working Group of the Paediatric Hemato-Oncology Italian Associations (AIEOP)

Common Variable Immunodeficiency (CVID) in Adults As First Manifestation of (cryptic) Dyskeratosis Congenita

Hyper-Sialylated IgG M254, an Innovative Therapeutic Candidate, Evaluated in Healthy Volunteers and in Patients with Immune Thrombocytopenia Purpura: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics

GATA2 -Dependent Developmental and Regenerative Networks

Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34 + Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major

Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia

Truncated Erythropoietin Receptors Confer an In Vivo Selective Advantage in Gene-Modified Erythroid Cells Expressing Fetal Hemoglobin Due to BCL11A Interference

Potential Influence of Hematological or Immunological Hereditary Predisposition Genes on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Hematological Malignancies

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

Activation of the Receptor Tyrosine Kinase (RET) By GDNF/GFRα1 Improves Cord Blood-Derived HSC in Vitro expansion and In Vivo engraftment

iPSC-Derived NK Cells Synergize with T Cells and Anti-PD-1 Antibody to Mediate Durable Anti-Tumor Responses In Vivo

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

Prophylactic Itacitinib (INCB039110) for the Prevention of Cytokine Release Syndrome Induced By Chimeric Antigen Receptor T-Cells (CAR-T-cells) Therapy

Secondary Autoimmune Neutropenia: Data from the Italian Neutropenia Registry

Vaccine Response to a Neo Polyssacharide Antigen Typhim Vi on the Identification of Secondary Immunodeficiencies in Hematological Malignancy

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American Society of Hematology

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