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Journal Articles

Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series

Blood (2024) 144 (16): 1699–1704.

https://doi.org/10.1182/blood.2024024524

Published: 2024

Includes: Supplemental data

Journal Articles

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Tanja C. Vallée, Jannik S. Glasmacher, Hannes Buchner, Peter D. Arkwright, Uta Behrends, Anastasia Bondarenko, Michael J. Browning, David Buchbinder, Alessandro Cattoni, Liudmyla Chernyshova, Peter Ciznar, Theresa Cole, Wojciech Czogała, Gregor Dueckers, John David M. Edgar, Fatih Erbey, Anders Fasth, Francesca Ferrua, Renata Formankova, Eleonora Gambineri, Andrew R. Gennery, Frederick D. Goldman, Luis I. Gonzalez-Granado, Carsten Heilmann, Tarja Heiskanen-Kosma, Hanna Juntti, Leena Kainulainen, Hirokazu Kanegane, Neslihan E. Karaca, Sara S. Kilic, Christoph Klein, Sylwia Kołtan, Irina Kondratenko, Isabelle Meyts, Gulnara M. Nasrullayeva, Lucia D. Notarangelo, Srdjan Pasic, Isabelle Pellier, Claudio Pignata, Siraj Misbah, Ansgar Schulz, Gesmar R. Segundo, Anna Shcherbina, Mary Slatter, Robert Sokolic, Pere Soler-Palacin, Polina Stepensky, Joris M. van Montfrans, Samppa Ryhänen, Beata Wolska-Kuśnierz, John B. Ziegler, Xiaodong Zhao, Alessandro Aiuti, Hans D. Ochs, Michael H. Albert

Blood (2024) 143 (24): 2504–2516.

https://doi.org/10.1182/blood.2023021411

Published: 2024

Includes: Supplemental data

Journal Articles

Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis

Blood (2024) 143 (19): 1937–1952.

https://doi.org/10.1182/blood.2023022666

Published: 2024

Includes: Supplemental data

Journal Articles

Unraveling Pathophysiology and Hematopoiesis of Vexas Syndrome By Multi-Omics Analyses and Targeted Gene Editing

Blood (2023) 142 (Supplement 1): 2692.

https://doi.org/10.1182/blood-2023-179749

Published: 2023

Journal Articles

Towards improved yet regulated gene therapy for X-CGD

Anna Kajaste-Rudnitski, Alessandro Aiuti

Blood (2023) 141 (9): 966–967.

https://doi.org/10.1182/blood.2022018800

Published: 2023

Journal Articles

Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

Blood (2022) 140 (Supplement 1): 1904–1905.

https://doi.org/10.1182/blood-2022-162195

Published: 2022

Journal Articles

Unveiling the Biological Role of Peripheral Blood Human Circulating Hematopoietic Stem and Progenitor Cells

Blood (2022) 140 (Supplement 1): 1977–1978.

https://doi.org/10.1182/blood-2022-163251

Published: 2022

Journal Articles

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Blood (2022) 139 (13): 2066–2079.

https://doi.org/10.1182/blood.2021014687

Published: 2022

Includes: Supplemental data

Journal Articles

Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency

Alessandra Mortellaro, Matteo Zoccolillo, Cristina Mesa Nuñez, Alessia Brix, Immacolata Brigida, Federica Barzaghi, Serena Scala, Raisa Jofra Hernández, Luca Basso-Ricci, Mariasilvia Colantuoni, Simone Cesaro, Francesca Conti, Andrea Pession, Fabio Benedetti, Marco Gattorno, Luigi Naldini, Maria Pia Cicalese, Anna Pistocchi, Alessandro Aiuti

Blood (2021) 138 (Supplement 1): 2937.

https://doi.org/10.1182/blood-2021-152127

Published: 2021

Journal Articles

Hematopoietic Stem Cells Are Endowed with Erythroid Signature in Beta-Thalassemia

Maria Rosa Lidonnici, PhD, Giulia Chianella, Francesca Tiboni, Matteo Barcella, PhD, Ivan Merelli, PhD, Samantha Scaramuzza, PhD, Claudia Rossi, Stefania Crippa, PhD, Maria Ester Bernardo, MD PhD, Fabio Ciceri, MD, Alessandro Aiuti, MD PhD, Sarah Marktel, MD, Giuliana Ferrari, PhD

Blood (2020) 136 (Supplement 1): 31.

https://doi.org/10.1182/blood-2020-137426

Published: 2020

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

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

T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency

Blood (2018) 132 (22): 2362–2374.

https://doi.org/10.1182/blood-2018-07-863431

Published: 2018

Includes: Supplemental data

Journal Articles

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Blood (2017) 130 (Supplement 1): 355.

https://doi.org/10.1182/blood.V130.Suppl_1.355.355

Published: 2017

Journal Articles

Impairment in the Mesenchymal Bone-Marrow Niche of Beta-Thalassemia Patients and Association with Prolonged Iron Exposure

Stefania Crippa, PhD, Valeria Rossella, Biologist, Laura Silvestri, PhD, Annamaria Aprile, PhD, Samantha Scaramuzza, Silvia Rivis, maria Antonietta Avanzini, Marco Zecca, MD, Sarah Marktel, MD, Fabio Ciceri, MD, Alessandro Aiuti, MD, Giuliana Ferrari, PhD, Maria Ester Bernardo, MD PhD

Blood (2017) 130 (Supplement 1): 948.

https://doi.org/10.1182/blood.V130.Suppl_1.948.948

Published: 2017

Journal Articles

Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications

Erika Zonari, PhD, Giacomo Desantis, PhD, Carolina Petrillo, Oriana Meo, Samantha Scaramuzza, PhD, Maria Rosa Lidonnici, PhD, Anna Kajaste-Rudnitski, PhD, Fabio Ciceri, MD, Sarah Marktel, Alessandro Aiuti, MD PhD, Giuliana Ferrari, PhD, Luigi Naldini, MD PhD, Bernhard Gentner, MD

Blood (2016) 128 (22): 4707.

https://doi.org/10.1182/blood.V128.22.4707.4707

Published: 2016

Journal Articles

Wiskott-Aldrich Syndrome: A Retrospective Study on 575 Patients Analyzing the Impact of Splenectomy, Stem Cell Transplantation, or No Definitive Treatment on Frequency of Disease-Related Complications and Physician-Perceived Quality of Life

Jannik S Glasmacher, Tanja C Bittner, MD, Hans D Ochs, MD, Alessandro Aiuti, MD PhD, Peter D Arkwright, MD, Dmitry Balashov, Uta Behrends, Bernd H. Belohradsky, MD, Elisa Bertoni, David K. Buchbinder, MD MSHS, Michael Browning, Anastasiia Bondarenko, Fabio Candotti, MD, Alessandro Cattoni, Liudmyla Chernyshova, Joseph H. Chewning, MD, Peter Ciznar, Theresa Cole, Beatriz T Costa-Carvalho, Wojciech Czogala, Gregor Dueckers, David M Edgar, Fatih Erbey, Anders Fasth, MD PhD, Renata Formankova, MD PhD, Tomas Freiberger, Eleonora Gambineri, Andrew Gennery, MD, Frederick D Goldman, MD, Luis I Gonzalez-Granado, Tiago N Gulmaraes, David Hagin, MD PhD, Fabian Hauck, MD PhD, Tarja Heiskanen-Kosma, Manfred Hoenig, MD, Hanna Juntti, Hirokazu Kanegane, Leena Kainulainen, Neslihan E Karaca, Sara S Kilic, Christoph Klein, MD PhD, Sylwia Koltan, Irina Kondratenko, Dawei Liu, MD, Susanne Matthes, MD, Juliana T L Mazzucchelli, Isabelle Meyts, Siraj Misbah, Zohreh Nademi, Gulnara Nasrullayeva, Lucia D Notarangelo, Pere Soler-Palacin, Olga Pashchenko, Srdjan Pasic, Isabelle Pellier, MD, Claudio Pignata, Camilla Roepstorff, Catharina Schuetz, Ansgar S Schulz, MD PhD Prof., Gesmar R S Segundo, Anna Shcherbina, Joanne Smart, Robert A. Sokolic, MD, Polina Stepensky, MD, Troy Torgerson, MD PhD, Svetlana Vakhlyarskaya, Joris van Montfrans, MD PhD, Kim Vettenranta, MD PD, Beata Wolska-Kusnierz, Xiaodong Zhao, John B Ziegler, Xuan Zhang, Michael H. Albert, MD

Blood (2016) 128 (22): 366.

https://doi.org/10.1182/blood.V128.22.366.366

Published: 2016

Journal Articles

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Blood (2016) 128 (1): 45–54.

https://doi.org/10.1182/blood-2016-01-688226

Published: 2016

Includes: Supplemental data

Journal Articles

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 259.

https://doi.org/10.1182/blood.V126.23.259.259

Published: 2015

Journal Articles

Tracking Genetically Engineered Lymphocytes Long-Term Reveals the Dynamics of T-Cell Immunological Memory

Blood (2015) 126 (23): 263.

https://doi.org/10.1182/blood.V126.23.263.263

Published: 2015

NARROW

Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis

Unraveling Pathophysiology and Hematopoiesis of Vexas Syndrome By Multi-Omics Analyses and Targeted Gene Editing

Towards improved yet regulated gene therapy for X-CGD

Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

Unveiling the Biological Role of Peripheral Blood Human Circulating Hematopoietic Stem and Progenitor Cells

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency

Hematopoietic Stem Cells Are Endowed with Erythroid Signature in Beta-Thalassemia

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

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

T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Impairment in the Mesenchymal Bone-Marrow Niche of Beta-Thalassemia Patients and Association with Prolonged Iron Exposure

Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications

Wiskott-Aldrich Syndrome: A Retrospective Study on 575 Patients Analyzing the Impact of Splenectomy, Stem Cell Transplantation, or No Definitive Treatment on Frequency of Disease-Related Complications and Physician-Perceived Quality of Life

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Tracking Genetically Engineered Lymphocytes Long-Term Reveals the Dynamics of T-Cell Immunological Memory

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American Society of Hematology

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American Society of Hematology

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