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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

Harnessing a Novel Dyrk1a-Ablim2-MKL1 Regulatory Module in Megakaryocyte Morphogenesis to Enable Scalable Platelet "Pharming"

Kamaleldin E Elagib, PhDMBBS, Ashton Brock, PhD, Goar Mosoyan, Cara Clementelli, Lorrie L Delehanty, BS, Alexandra Pacheco-Benichou, Corinne Fruit, Thierry Besson, PhD, Stephen Morris, MD, Koji Eto, MD, Camelia Iancu-Rubin, PhD, Adam N Goldfarb, MD

Blood (2019) 134 (Supplement_1): 3250.

https://doi.org/10.1182/blood-2019-129049

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

RAC1 Inhibitor EHT1864 and Venetoclax Overcome Midostaurin Resistance in Acute Myeloid Leukemia

Blood (2019) 134 (Supplement_1): 1277.

https://doi.org/10.1182/blood-2019-129762

Published: 2019

Journal Articles

Wave-Complex-Dependent Actin Dynamics Controls Hematopoietic Stem Cell Bone Marrow Retention and Orderly Differentiation into Mature Hematopoietic Lineages

Eliza Wiercinska, PhD, David de Gorter, PhD, Eva Danner, Sabine Harenkamp, Andre Gorgens, PhD, Anja Schwiebs, PhD, Theresia Stradal, PhD, Halvard Bonig, MD MA

Blood (2017) 130 (Supplement 1): 2417.

https://doi.org/10.1182/blood.V130.Suppl_1.2417.2417

Published: 2017

Journal Articles

The WASP P460S Mutation Causes a New Phenotype of WASP Mutations Related Disorder: X-Linked Pancytopenia

Yunjing Zheng, MD, Shuiyan Wu, Xiaoliang Yu, Meng Wu, Qin Lu, Sudan He, Shaoyan Hu, MD PhD

Blood (2017) 130 (Supplement 1): 1044.

https://doi.org/10.1182/blood.V130.Suppl_1.1044.1044

Published: 2017

Journal Articles

A Novel Mutant Wasp in X-Linked Neutropenia Partially Recapitulates Defective Neutrophil Production through Its Expression in WASP-Deficient NB4 Cells

Masayuki Kobayashi, MD, Bidisha Chanda, MD PhD, Muneyoshi Futami, MD PhD, Mizuho Yamashita, Mitsuhito Hirano, MD, Kazuaki Yokoyama, MD PhD, Yoichi Imai, MDPhD, Eigo Shimizu, Nozomi Yusa, Rui Yamaguchi, PhD, Seiya Imoto, PhD, Satoru Miyano, PhD, Arinobu Tojo, MD PhD

Blood (2017) 130 (Supplement 1): 3560.

https://doi.org/10.1182/blood.V130.Suppl_1.3560.3560

Published: 2017

Journal Articles

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Blood (2017) 130 (11): 1327–1335.

https://doi.org/10.1182/blood-2017-04-777136

Published: 2017

Includes: Supplemental data

Journal Articles

Optimization of CSF3R Mutation Detection in Severe Congenital Neutropenia and Cyclic Neutropenia Patients for Routine Diagnostics Using Next Generation Sequencing

Maksim Klimiankou, PhD, Cornelia Zeidler, MD, Sabine Mellor-Heineke, Ingeborg Steiert, Karin Haehnel, Karl Welte, MD PhD, Julia Skokowa, MD PhD

Blood (2016) 128 (22): 3685.

https://doi.org/10.1182/blood.V128.22.3685.3685

Published: 2016

Journal Articles

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

Adrian Schwarzer, MD PhD, Steven R Talbot, PhD, Oliver Dittrich-Breiholz, PhD, Adrian J Thrasher, MD PhD, Bobby Gaspar, MD, Ute Modlich, Christopher Baum, MD, Axel Schambach, MD PhD, Michael Rothe, PhD

Blood (2016) 128 (22): 4710.

https://doi.org/10.1182/blood.V128.22.4710.4710

Published: 2016

Journal Articles

The early activation of memory B cells from Wiskott-Aldrich syndrome patients is suppressed by CD19 downregulation

Xiaoming Bai, Yongjie Zhang, Lu Huang, Jinzhi Wang, Wenyan Li, Linlin Niu, Hongyan Jiang, Rongxin Dai, Lina Zhou, Zhiyong Zhang, Heather Miller, Wenxia Song, Xiaodong Zhao, Chaohong Liu

Blood (2016) 128 (13): 1723–1734.

https://doi.org/10.1182/blood-2016-03-703579

Published: 2016

Journal Articles

Abnormalities of follicular helper T-cell number and function in Wiskott-Aldrich syndrome

Xuan Zhang, Rongxin Dai, Wenyan Li, Hongyi Zhao, Yongjie Zhang, Lina Zhou, Hongqiang Du, Guangjin Luo, Junfeng Wu, Linlin Niu, Yunfei An, Zhiyong Zhang, Yuan Ding, Wenxia Song, Chaohong Liu, Xiaodong Zhao

Blood (2016) 127 (25): 3180–3191.

https://doi.org/10.1182/blood-2015-06-652636

Published: 2016

Includes: Supplemental data

Journal Articles

The sting of WASP deficiency: autoimmunity exposed

Andrew R. Gennery

Blood (2016) 127 (2): 173–175.

https://doi.org/10.1182/blood-2015-10-677237

Published: 2016

Journal Articles

N-WASP is required for B-cell–mediated autoimmunity in Wiskott-Aldrich syndrome

Stefano Volpi, Elettra Santori, Katrina Abernethy, Masayuki Mizui, Carin I. M. Dahlberg, Mike Recher, Kelly Capuder, Eva Csizmadia, Douglas Ryan, Divij Mathew, George C. Tsokos, Scott Snapper, Lisa S. Westerberg, Adrian J. Thrasher, Fabio Candotti, Luigi D. Notarangelo

Blood (2016) 127 (2): 216–220.

https://doi.org/10.1182/blood-2015-05-643817

Published: 2016

Includes: Supplemental data

Journal Articles

Dynamin 2 (DNM2) and PACSIN2 Regulate Megakaryocyte Demarcation Membrane System Formation and Platelet Production in Concert

Silvia Giannini, PhD, Markus Bender, PhD, Fred G. Pluthero, PhD, Hilary Christensen, PhD, Richard Leung, PhD, Richard W. Lo, PhD, Jan Kormann, PhD, Markus Plomann, PhD, Walter H.A. Kahr, MD PhD, Hervé Falet, PhD

Blood (2015) 126 (23): 419.

https://doi.org/10.1182/blood.V126.23.419.419

Published: 2015

Journal Articles

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 260.

https://doi.org/10.1182/blood.V126.23.260.260

Published: 2015

Journal Articles

ARHGAP21 Is Upregulated and Triggers the Modulation of Rho Gtpase Signaling Pathways during Megakaryocytic Differentiation

Vanessa Aline Bernusso, Mariana Lazarini, PhD, João Agostinho Machado-Neto, PhD, Karin Spat Albino Barcellos, PhD, Sara Teresinha Olalla Saad, PhD MD

Blood (2015) 126 (23): 4760.

https://doi.org/10.1182/blood.V126.23.4760.4760

Published: 2015

Journal Articles

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

Alessandra Magnani, MD PhD, Cécile Roudaut, MSc, Aurelie Gabrion, Laure Caccavelli, PhD, Fabien Touzot, MDPhD, Benedicte Neven, MD PhD, Despina Moshous, MD, Nizar Mahlaoui, Emmanuel Clave, Antoine Toubert, Alain Fischer, MD PhD, Marina Cavazzana, MD PhD

Blood (2015) 126 (23): 3235.

https://doi.org/10.1182/blood.V126.23.3235.3235

Published: 2015

Journal Articles

SUMOylation-disrupting WAS mutation converts WASp from a transcriptional activator to a repressor of NF-κB response genes in T cells

Koustav Sarkar, Sanjoy Sadhukhan, Seong-Su Han, Yatin M. Vyas

Blood (2015) 126 (14): 1670–1682.

https://doi.org/10.1182/blood-2015-05-646182

Published: 2015

Includes: Supplemental data

Journal Articles

HIV-1 reprograms the migration of macrophages

Christel Vérollet, Shanti Souriant, Emilie Bonnaud, Paul Jolicoeur, Brigitte Raynaud-Messina, Cassandre Kinnaer, Isabelle Fourquaux, Andrea Imle, Serge Benichou, Oliver T. Fackler, Renaud Poincloux, Isabelle Maridonneau-Parini

Blood (2015) 125 (10): 1611–1622.

https://doi.org/10.1182/blood-2014-08-596775

Published: 2015

Includes: Multimedia, Supplemental data

NARROW

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

Harnessing a Novel Dyrk1a-Ablim2-MKL1 Regulatory Module in Megakaryocyte Morphogenesis to Enable Scalable Platelet "Pharming"

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

RAC1 Inhibitor EHT1864 and Venetoclax Overcome Midostaurin Resistance in Acute Myeloid Leukemia

Wave-Complex-Dependent Actin Dynamics Controls Hematopoietic Stem Cell Bone Marrow Retention and Orderly Differentiation into Mature Hematopoietic Lineages

The WASP P460S Mutation Causes a New Phenotype of WASP Mutations Related Disorder: X-Linked Pancytopenia

A Novel Mutant Wasp in X-Linked Neutropenia Partially Recapitulates Defective Neutrophil Production through Its Expression in WASP-Deficient NB4 Cells

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Optimization of CSF3R Mutation Detection in Severe Congenital Neutropenia and Cyclic Neutropenia Patients for Routine Diagnostics Using Next Generation Sequencing

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

The early activation of memory B cells from Wiskott-Aldrich syndrome patients is suppressed by CD19 downregulation

Abnormalities of follicular helper T-cell number and function in Wiskott-Aldrich syndrome

The sting of WASP deficiency: autoimmunity exposed

N-WASP is required for B-cell–mediated autoimmunity in Wiskott-Aldrich syndrome

Dynamin 2 (DNM2) and PACSIN2 Regulate Megakaryocyte Demarcation Membrane System Formation and Platelet Production in Concert

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

ARHGAP21 Is Upregulated and Triggers the Modulation of Rho Gtpase Signaling Pathways during Megakaryocytic Differentiation

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

SUMOylation-disrupting WAS mutation converts WASp from a transcriptional activator to a repressor of NF-κB response genes in T cells

HIV-1 reprograms the migration of macrophages

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American Society of Hematology

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American Society of Hematology

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