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Journal Articles

Zhang X, Dia R, Li W, et al. Abnormalities of follicular helper T-cell number and function in Wiskott-Aldrich syndrome. Blood. 2016;127(25):3180-3191.

Blood (2020) 135 (24): 2198.

https://doi.org/10.1182/blood.2020006609

Published: 2020

Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

Journal Articles

Bai X, Zhang Y, Huang L, et al. The early activation of memory B cells from Wiskott-Aldrich syndrome patients is suppressed by CD19 downregulation. Blood . 2016;128(13):1723-1734.

Blood (2019) 134 (22): 1997.

https://doi.org/10.1182/blood.2019003731

Published: 2019

Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

B-Cell Maturation Antigen (BCMA) in Systemic Light-Chain Amyloidosis (AL): Association with Disease Activity and Its Modulation with Gamma-Secretase Inhibition

Amandeep Godara, MD, Ping Zhou, MD PhD, Benjamin Rosenthal, BS, Adin Kugelmass, BSc, Denis Toskic, BS, Teresa Fogaren, NP, Cindy Varga, MD BSc, Raymond L Comenzo, MD

Blood (2019) 134 (Supplement_1): 4409.

https://doi.org/10.1182/blood-2019-126988

Published: 2019

Journal Articles

Gastrointestinal Plasmoblastic Lymphoma Associated with Wiskott-Aldrich Syndrome: A Case Report

Ali Unal, MD, Serhat Çelik, Zeynep Güven, MD, Leylagul Kaynar, MD, Bulent Eser, MD, Mustafa Çetin

Blood (2019) 134 (Supplement_1): 5359.

https://doi.org/10.1182/blood-2019-123387

Published: 2019

Journal Articles

Receptor Tyrosine Kinase AXL: A Potential Strategy to Counter Immune Suppression and Dormancy in Multiple Myeloma

Kim De Veirman, Siyang Yan, Ken Maes, Nathan De Beule, MD, Sylvia Faict, MSc, Elke De Bruyne, PhD, Eline Menu, PhD, Karin Vanderkerken, PhD

Blood (2019) 134 (Supplement_1): 4335.

https://doi.org/10.1182/blood-2019-126834

Published: 2019

Journal Articles

Proteomic Analysis Identifies Mechanism(s) of Overcoming Bortezomib-Resistance Via Targeting Ubiquitin Receptor Rpn13

Ting Du, PhD, Yan Song, PhD, Arghya Ray, PhD, Dharminder Chauhan, PhD, Kenneth C. Anderson, MD

Blood (2019) 134 (Supplement_1): 3074.

https://doi.org/10.1182/blood-2019-130236

Published: 2019

Journal Articles

Overexpression and Targeted Activation of the Protein Phosphatases SHP-1 Abrogates Survival Pathways in Large Granular Lymphocyte Leukemia (LGLL)

Mario Angelo Pagano, Elena Tibaldi, Giulia Calabretto, Antonella Teramo, Luca Dell'Amico, Gabriella Donà, Gregorio Barilà, Cristina Vicenzetto, Vanessa Rebecca Gasparini, Valentina Trimarco, Monica Facco, Francesco Piazza, Gianpietro Semenzato, Anna Maria Brunati, Renato Zambello

Blood (2019) 134 (Supplement_1): 2798.

https://doi.org/10.1182/blood-2019-129883

Published: 2019

Journal Articles

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Bernhard Gentner, MD, Francesca Farina, MD, Matteo Giovanni Carrabba, MD, Magda Marcatti, MD, Stefania Girlanda, MD, Gabriele Antonarelli, MD, Capotondo Alessia, PhD, Stefania Mazzoleni, PhD, Carlo Russo, MD, Luigi Naldini, MD PhD, Fabio Ciceri, MD

Blood (2019) 134 (Supplement_1): 2064.

https://doi.org/10.1182/blood-2019-123749

Published: 2019

Journal Articles

YAP and TAZ in Fibroblastic Reticular Cells Support Hematopoiesis and Retention of Lymphocytes in Lymph Nodes

Hyunsoo Cho, MD PhD, Yu Ri Kim, MD, Jieun Jang, MD, Haerim Chung, MD, Soo-Jeong Kim, Sung Yong Choi, Bae Hosung, Ralf Adams, Burkhard Ludewig, PhD, Jin Seok Kim, MD PhD, June-Won Cheong, MD PhD, Yoo Hong Min, MD PhD, Gou Young Koh

Blood (2019) 134 (Supplement_1): 3595.

https://doi.org/10.1182/blood-2019-124625

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

Published: 2019

Journal Articles

Sickle Cell Hemoglobin Impairs Lysosomal Processing in Macrophages Leading to Inflammasome Activation and Inflammation

Marina Jerebtsova, PhD, Xiaomei Niu, MD, Sharmin Diaz, NP, Namita Kumari, PhD, Sergei Nekhai, PhD

Blood (2019) 134 (Supplement_1): 979.

https://doi.org/10.1182/blood-2019-130741

Published: 2019

Journal Articles

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Blood (2019) 134 (Supplement_1): 3346.

https://doi.org/10.1182/blood-2019-124665

Published: 2019

Journal Articles

Platelet and Immune Characteristics of Patients with Immune Thrombocytopaenia Non Responders to Therapeutic Treatments

Elena Monzón Manzano, Raul Justo Sanz, Diana Hernández, Teresa Álvarez Roman, Ihosvany Fernandez-Bello, Mónica Martín, Larissa Valor, María Isabel Rivas Pollmar, Miguel A Canales, MD PhD, Victor Jimenez-Yuste, Nora Butta, PhD

Blood (2019) 134 (Supplement_1): 1089.

https://doi.org/10.1182/blood-2019-125772

Published: 2019

Journal Articles

Increased STAT5/STAT3 Intracellular Signaling in Circulating CD34+ Cells of Patients with PMF Correlates with Disease Severity

Carlotta Abbà, MSc, Rita Campanelli, Paolo Catarsi, Laura Villani, Vittorio Abbonante, PhD, Christian Andrea Di Buduo, PhD, Melania Antonietta Sesta, Vittorio Rosti, MD, Giovanni Barosi, MD, Margherita Massa

Blood (2018) 132 (Supplement 1): 4337.

https://doi.org/10.1182/blood-2018-99-114684

Published: 2018

Journal Articles

Results of a Prospective, Multicenter, Phase I/II Clinical Study in Pediatric and Adult Patients Using TCR Alpha/Beta and CD19 Depleted Haploidentical Hematopoietic Stem Cell Grafts Following Reduced-Intensity Conditioning

Blood (2018) 132 (Supplement 1): 604.

https://doi.org/10.1182/blood-2018-99-110124

Published: 2018

Journal Articles

Reduction of Graft Rejection By Fludarabine Combined Busulfan, Cyclophosphamide and Antithymocyte Globulin Conditioning in Cord Blood Transplantation for Children with Wiskott-Aldrich Syndrome

Peifang Xiao, MD, Jun Lu, MD, Jie Li, MD, Defei Zheng, MD, Li Gao, MD, Ailian Guo, MD, Hu Liu, MD, Suxiang Liu, MD, Hailong He, MD, Shaoyan Hu, MD PhD

Blood (2018) 132 (Supplement 1): 5714.

https://doi.org/10.1182/blood-2018-99-117748

Published: 2018

Journal Articles

The Related Factors and the Prognosis of Nervous System Complications on the Pediatric Patients with Hematopoietic Stem Cell Transplantation

Suxiang Liu, MD, Defei Zheng, MD, Peifang Xiao, MD, Yi Wang, MD, Zong Zhai, MD, Jun Lu, MD, Jie Li, MD, Shaoyan Hu, MD PhD

Blood (2018) 132 (Supplement 1): 5718.

https://doi.org/10.1182/blood-2018-99-117803

Published: 2018

Journal Articles

Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen in Patients with Wiskott-Aldrich Syndrome Given Allogeneic Hematopoietic Cell Transplantation — an EBMT Inborn Errors Working Party and Scetide Retrospective Analysis

Blood (2018) 132 (Supplement 1): 2175.

https://doi.org/10.1182/blood-2018-99-112394

Published: 2018

NARROW

Zhang X, Dia R, Li W, et al. Abnormalities of follicular helper T-cell number and function in Wiskott-Aldrich syndrome. Blood. 2016;127(25):3180-3191.

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Bai X, Zhang Y, Huang L, et al. The early activation of memory B cells from Wiskott-Aldrich syndrome patients is suppressed by CD19 downregulation. Blood . 2016;128(13):1723-1734.

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

B-Cell Maturation Antigen (BCMA) in Systemic Light-Chain Amyloidosis (AL): Association with Disease Activity and Its Modulation with Gamma-Secretase Inhibition

Gastrointestinal Plasmoblastic Lymphoma Associated with Wiskott-Aldrich Syndrome: A Case Report

Receptor Tyrosine Kinase AXL: A Potential Strategy to Counter Immune Suppression and Dormancy in Multiple Myeloma

Proteomic Analysis Identifies Mechanism(s) of Overcoming Bortezomib-Resistance Via Targeting Ubiquitin Receptor Rpn13

Overexpression and Targeted Activation of the Protein Phosphatases SHP-1 Abrogates Survival Pathways in Large Granular Lymphocyte Leukemia (LGLL)

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

YAP and TAZ in Fibroblastic Reticular Cells Support Hematopoiesis and Retention of Lymphocytes in Lymph Nodes

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Sickle Cell Hemoglobin Impairs Lysosomal Processing in Macrophages Leading to Inflammasome Activation and Inflammation

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Platelet and Immune Characteristics of Patients with Immune Thrombocytopaenia Non Responders to Therapeutic Treatments

Increased STAT5/STAT3 Intracellular Signaling in Circulating CD34+ Cells of Patients with PMF Correlates with Disease Severity

Results of a Prospective, Multicenter, Phase I/II Clinical Study in Pediatric and Adult Patients Using TCR Alpha/Beta and CD19 Depleted Haploidentical Hematopoietic Stem Cell Grafts Following Reduced-Intensity Conditioning

Reduction of Graft Rejection By Fludarabine Combined Busulfan, Cyclophosphamide and Antithymocyte Globulin Conditioning in Cord Blood Transplantation for Children with Wiskott-Aldrich Syndrome

The Related Factors and the Prognosis of Nervous System Complications on the Pediatric Patients with Hematopoietic Stem Cell Transplantation

Busulfan/Fludarabine- or Treosulfan/Fludarabine-Based Conditioning Regimen in Patients with Wiskott-Aldrich Syndrome Given Allogeneic Hematopoietic Cell Transplantation — an EBMT Inborn Errors Working Party and Scetide Retrospective Analysis

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American Society of Hematology

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American Society of Hematology

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