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Journal Articles

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

Malte U Ritter, Benjamin Secker, Maksim Klimiankou, Masoud Nasri, MSc, Narges Aghaallaei, Benjamin Dannenmann, Regine Bernhard, Ingeborg Steiert, Karin Haehnel, Baubak Bajoghli, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 1036.

https://doi.org/10.1182/blood-2019-131708

Published: 2019

Journal Articles

102 Patients with Primary Autoimmune Neutropenia : An Analysis of the European Severe Chronic Neutropenia International Registry (SCNIR)

Fabian Frömling, Sabine Mellor-Heineke, Natali Gerschmann, Lena Röbbel, Julia Skokowa, MD PhD, Karl Welte, Cornelia Zeidler

Blood (2019) 134 (Supplement_1): 1039.

https://doi.org/10.1182/blood-2019-130031

Published: 2019

Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

Published: 2019

Journal Articles

Splenic CD24 low Red Pulp Macrophages Provide an Alternate Niche for Chronic Myeloid Leukemia Stem Cells

Michael A Amrein, MD PhD, Elias D Bührer, MD PhD, Stefan Forster, MD, Stephan Isringhausen, Christian M Schürch, MD PhD, Salil S. Bhate, MMath, Mohamad Al Sayed, PhD, Cesar Nombela Arrieta, PhD, Ramin Radpour, PhD, Carsten Riether, PhD, Adrian F Ochsenbein, MD

Blood (2019) 134 (Supplement_1): 1634.

https://doi.org/10.1182/blood-2019-126593

Published: 2019

Journal Articles

Activity of Venetoclax-Based Therapy in Myelodysplastic Syndrome (MDS)

Jorge Cortes, MD, Guillermo Garcia-Manero, MD, Koji Sasaki, MD, Kiran Naqvi, MDMPH, Yesid Alvarado, MD, Tapan M. Kadia, MD, Farhad Ravandi, MD, Jorge E. Cortes, MD, Naval G. Daver, MD, Koichi Takahashi, MD, Elias Jabbour, MD, Gautam M. Borthakur, MD, Naveen Pemmaraju, MD, Marina Y Konopleva, MD PhD, Sherry A. Pierce, BSN, BA, Carlos E. Bueso-Ramos, MD PhD, Keyur P. Patel, MBBS, PhD, Hagop M. Kantarjian, MD, Guillermo Montalban Bravo, MD

Blood (2019) 134 (Supplement_1): 1726.

https://doi.org/10.1182/blood-2019-126005

Published: 2019

Journal Articles

Randomized Open-Labeled Academic Trial Comparing Standard AZA Therapy with Combination of G-CSF with AZA in High Risk MDS Patients - Interim Analysis

Anna Jonasova, MD PhD, Lubomir Minarik, MD, Vojtech Kulvait, PhD, Michal Pesta, PhD, Adel Schaffartzikova, MD, Galina Kislik, PhD, Nina Dusilkova, MD, Zuzana Zemanova, PhD, Tomas Stopka, MD PhD

Blood (2019) 134 (Supplement_1): 1729.

https://doi.org/10.1182/blood-2019-124274

Published: 2019

Journal Articles

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Jean-Sebastien Diana, MD, Sandra Manceau, PhD, Thierry Leblanc, MD, Chloé Couzin, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Matthieux Bendavid, MD, Laure Joseph, MD, Marianne Delville, MD, Stephane Blanche, MD, Jean Soulier, MD PhD, Marina Cavazzana, MD PhD, Francois Lefrere, MD

Blood (2019) 134 (Supplement_1): 1970.

https://doi.org/10.1182/blood-2019-128051

Published: 2019

Journal Articles

Megakaryocyte Reprogramming in Breast Cancer

Harvey G. Roweth, BSc, PhD, Michael Malloy, BS, Gregory Goreczny, BS, PhD, Kelly E. Johnson, BS, PhD, Sandra S. McAllister, PhD, Joseph E. Italiano, Jr., BS, PhD, Elisabeth Battinelli, MD PhD

Blood (2019) 134 (Supplement_1): 12.

https://doi.org/10.1182/blood-2019-124894

Published: 2019

Journal Articles

Targeting CXCR4, VLA4, and CXCR2 for Hematopoietic Stem Cell Mobilization

Daniel Cancilla, BS, Haresh Thakellapalli, Marvin J Meyers, PhD, Michael P. Rettig, PhD, Ezhilarasi Chendamarai, PhD, Stephanie Christ, MS, Darja Karpova, PhD, Peter G Ruminski, BS, Julie K. Ritchey, Dwight M. Morrow, PhD, Michael Prinsen, John F. DiPersio, MD PhD

Blood (2019) 134 (Supplement_1): 1916.

https://doi.org/10.1182/blood-2019-124373

Published: 2019

Journal Articles

Excellent Survival and Immune Reconstitution Following Matched/Mismatched Unrelated and Mismatched Related Transplantation in Adult Patients with Sickle Cell Disease: Updated Results from a Single Center Experience

Amer Assal, MD, Diane George, MD, Monica Bhatia, MD, Divaya Bhutani, MD, Christian Gordillo, BS, Suzanne Lentzsch, MD PhD, Ran Reshef, MDMSc, Markus Y Mapara, MD PhD

Blood (2019) 134 (Supplement_1): 2043.

https://doi.org/10.1182/blood-2019-132113

Published: 2019

Journal Articles

A Randomized Evaluation of Vinorelbine Versus Gemcitabine Chemotherapy Mobilization of Stem Cells in Myeloma Patients

Barbara Jeker, Sarah Farag, Urban Novak, Ulrike Bacher, Behrouz Mansouri Taleghani, Qiyu Li, Thomas Pabst, MD

Blood (2019) 134 (Supplement_1): 1963.

https://doi.org/10.1182/blood-2019-127848

Published: 2019

Journal Articles

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Alexis Leonard, MD, Naoya Uchida, MD, David F. Stroncek, MD, Sandhya R. Panch, MDMPH, Kamille West, Eoghan George Molloy, MD, Tom Hughes, Pharm.D., Sara Hauffe, RN, Tiffani Taylor, Courtney D. Fitzhugh, MD, Jane S. Hankins, MD MS, Megan Wilson, MSW, CCRP, Akshay Sharma, MBBS, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Matthew Hsieh, MD, John F. Tisdale, MD

Blood (2019) 134 (Supplement_1): 1964.

https://doi.org/10.1182/blood-2019-126091

Published: 2019

Journal Articles

Severe Chronic Neutropenia in the Large Granular Lymphocyte Syndrome: Outcomes in Response to Granulocyte Colony Stimulating Factor (G-CSF) and Immunosuppressive Therapies

David C. Dale, MD, James A. Shannon, IV, Audrey Anna Bolyard, RN, BS, Jim Connelly, MD, Daniel C. Link, MD, Mary Ann Bonilla, MD, Peter E. Newburger, MD

Blood (2019) 134 (Supplement_1): 3589.

https://doi.org/10.1182/blood-2019-126181

Published: 2019

Journal Articles

A Phase I Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Tripegfilgrastim (Dulastin®) after Single Administration in Pediatric Patients

Kyung Taek Hong, MD, Soyoung Lee, Jaeseong Oh, Jung Yoon Choi, MD, Hee Young Shin, MD PhD, Kyung-Sang Yu, MD PhD, Hyoung Jin Kang, MD PhD

Blood (2019) 134 (Supplement_1): 3594.

https://doi.org/10.1182/blood-2019-125266

Published: 2019

Journal Articles

Quality of Life and Patient Reported Outcomes in Severe Chronic Neutropenia

Thomas F. Michniacki, MD, Lauren E. Merz, MDMSc, Harlan McCaffrey, MA MS, James A. Connelly, MD, Kelly J. Walkovich, MD

Blood (2019) 134 (Supplement_1): 3449.

https://doi.org/10.1182/blood-2019-126808

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

Granulocyte Transfusions in a Cohort of Neutropenic Patients with Life-Threatening Infections and Hematologic Diseases

Asiri Ediriwickrema, MD, Mrigender Virk, MD, Jennifer Andrews, MDMSc

Blood (2019) 134 (Supplement_1): 3696.

https://doi.org/10.1182/blood-2019-127103

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

Phase 1b Study of Vipor (Venetoclax, Ibrutinib, Prednisone, Obinutuzumab, and Lenalidomide) in Relapsed/Refractory B-Cell Lymphoma: Safety, Efficacy and Molecular Analysis

Christopher Melani, MD, Rahul Lakhotia, MD, Stefania Pittaluga, MD PhD, Milos D. Miljkovic, MDMSc, Jagan R. Muppidi, MD PhD, Craig A. Portell, MD, Rafic Farah, MD, Seung Tae Lee, MD PhD, Anna Marie Juanitez, RN, Lydia L. Chou, CRNP, Seth M. Steinberg, PhD, Elaine S. Jaffe, MD, Louis Staudt, MD, Mark Roschewski, MD, Wyndham Wilson, MD

Blood (2019) 134 (Supplement_1): 2867.

https://doi.org/10.1182/blood-2019-125990

Published: 2019

Journal Articles

The Impact of Different Mobilization Strategies in the Setting of Multiple Myeloma

Francesco Mazziotta, MD, Gabriele Buda, MD PhD, Nadia Cecconi, MD, Giulia Cervetti, MD, Lorenzo Iovino, MD, Enrico Orciuolo, MD PhD, Matteo Pelosini, MD PhD, Edoardo Benedetti, MD, Francesco Caracciolo, MD, Giovanni Carulli, MD, Sara Galimberti, MD PhD, Mario Petrini, MD PhD

Blood (2019) 134 (Supplement_1): 3254.

https://doi.org/10.1182/blood-2019-126118

Published: 2019

NARROW

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

102 Patients with Primary Autoimmune Neutropenia : An Analysis of the European Severe Chronic Neutropenia International Registry (SCNIR)

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Splenic CD24 low Red Pulp Macrophages Provide an Alternate Niche for Chronic Myeloid Leukemia Stem Cells

Activity of Venetoclax-Based Therapy in Myelodysplastic Syndrome (MDS)

Randomized Open-Labeled Academic Trial Comparing Standard AZA Therapy with Combination of G-CSF with AZA in High Risk MDS Patients - Interim Analysis

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Megakaryocyte Reprogramming in Breast Cancer

Targeting CXCR4, VLA4, and CXCR2 for Hematopoietic Stem Cell Mobilization

Excellent Survival and Immune Reconstitution Following Matched/Mismatched Unrelated and Mismatched Related Transplantation in Adult Patients with Sickle Cell Disease: Updated Results from a Single Center Experience

A Randomized Evaluation of Vinorelbine Versus Gemcitabine Chemotherapy Mobilization of Stem Cells in Myeloma Patients

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Severe Chronic Neutropenia in the Large Granular Lymphocyte Syndrome: Outcomes in Response to Granulocyte Colony Stimulating Factor (G-CSF) and Immunosuppressive Therapies

A Phase I Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Tripegfilgrastim (Dulastin®) after Single Administration in Pediatric Patients

Quality of Life and Patient Reported Outcomes in Severe Chronic Neutropenia

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Granulocyte Transfusions in a Cohort of Neutropenic Patients with Life-Threatening Infections and Hematologic Diseases

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

Phase 1b Study of Vipor (Venetoclax, Ibrutinib, Prednisone, Obinutuzumab, and Lenalidomide) in Relapsed/Refractory B-Cell Lymphoma: Safety, Efficacy and Molecular Analysis

The Impact of Different Mobilization Strategies in the Setting of Multiple Myeloma

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American Society of Hematology

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American Society of Hematology

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