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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

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

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

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

Nicholas Hubbard, David Hagin, Karen Sommer, Yumei Song, Iram Khan, Courtnee Clough, Hans D. Ochs, David J. Rawlings, Andrew M. Scharenberg, Troy R. Torgerson

Blood (2016) 127 (21): 2513–2522.

https://doi.org/10.1182/blood-2015-11-683235

Published: 2016

Includes: Supplemental data

Journal Articles

Accumulation of peripheral autoreactive B cells in the absence of functional human regulatory T cells

Tuure Kinnunen, Nicolas Chamberlain, Henner Morbach, Jinyoung Choi, Sangtaek Kim, Joseph Craft, Lloyd Mayer, Caterina Cancrini, Laura Passerini, Rosa Bacchetta, Hans D. Ochs, Troy R. Torgerson, Eric Meffre

Blood (2013) 121 (9): 1595–1603.

https://doi.org/10.1182/blood-2012-09-457465

Published: 2013

Includes: Supplemental data

Journal Articles

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

Alexander Astrakhan, Blythe D. Sather, Byoung Y. Ryu, Socheath Khim, Swati Singh, Stephanie Humblet-Baron, Hans D. Ochs, Carol H. Miao, David J. Rawlings

Blood (2012) 119 (19): 4395–4407.

https://doi.org/10.1182/blood-2011-03-340711

Published: 2012

Includes: Supplemental data

Journal Articles

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

Blood (2011) 118 (6): 1675–1684.

https://doi.org/10.1182/blood-2010-11-319376

Published: 2011

Includes: Supplemental data

Journal Articles

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

Blood (2010) 115 (16): 3231–3238.

https://doi.org/10.1182/blood-2009-09-239087

Published: 2010

Includes: Supplemental data

Journal Articles

Anti-CD3 antibodies modulate anti–factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy

Baowei Peng, Peiqing Ye, David J. Rawlings, Hans D. Ochs, Carol H. Miao

Blood (2009) 114 (20): 4373–4382.

https://doi.org/10.1182/blood-2009-05-217315

Published: 2009

Includes: Supplemental data

Journal Articles

CD4 + FOXP3 + regulatory T cells confer long-term regulation of factor VIII–specific immune responses in plasmid-mediated gene therapy–treated hemophilia mice

Carol H. Miao, Benjamin R. Harmeling, Steven F. Ziegler, Benjamin C. Yen, Troy Torgerson, Liping Chen, Roger J. Yau, Baowei Peng, Arthur R. Thompson, Hans D. Ochs, David J. Rawlings

Blood (2009) 114 (19): 4034–4044.

https://doi.org/10.1182/blood-2009-06-228155

Published: 2009

Includes: Supplemental data

Journal Articles

Relevance of biallelic versus monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes

Blood (2009) 113 (9): 1967–1976.

https://doi.org/10.1182/blood-2008-02-141937

Published: 2009

Includes: Supplemental data

Journal Articles

Clinical Phenotype and Long Term Outcome in a Large Cohort of X-Linked Thrombocytopenia (XLT)/Mild Wiskott-Aldrich-Syndrome Patients

Blood (2008) 112 (11): 90.

https://doi.org/10.1182/blood.V112.11.90.90

Published: 2008

Journal Articles

WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function

Lisa S. Westerberg, Miguel A. de la Fuente, Fredrik Wermeling, Hans D. Ochs, Mikael C. I. Karlsson, Scott B. Snapper, Luigi D. Notarangelo

Blood (2008) 112 (10): 4139–4147.

https://doi.org/10.1182/blood-2008-02-140715

Published: 2008

Includes: Supplemental data

Journal Articles

Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice

Baowei Peng, Peiqing Ye, Bruce R. Blazar, Gordon J. Freeman, David J. Rawlings, Hans D. Ochs, Carol H. Miao

Blood (2008) 112 (5): 1662–1672.

https://doi.org/10.1182/blood-2008-01-128413

Published: 2008

Journal Articles

Transient Blockade of ICOS Co-Stimulatory Pathways Induces Long-Term Tolerance to Factor VIII Following Nonviral Gene Transfer of Factor VIII Plasmid into Hemophilia A Mice.

Baowei Peng, PhD, Peiqing Ye, MD, Bruce R. Blazer, MD, Hans D. Ochs, MD, MSc, Carol H. Miao, PhD

Blood (2006) 108 (11): 3283.

https://doi.org/10.1182/blood.V108.11.3283.3283

Published: 2006

Journal Articles

Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice

Carol H. Miao, Peiqing Ye, Arthur R. Thompson, David J. Rawlings, Hans D. Ochs

Blood (2006) 108 (1): 19–27.

https://doi.org/10.1182/blood-2005-11-4532

Published: 2006

Journal Articles

Postgrafting Immune Suppression Combined with Nonmyeloablative Conditioning for Transplantation of HLA-Identical Hematopoietic Cell Grafts: Results of a Phase I Study for Treatment of Immunodeficiency Disorders.

Lauri Burroughs, MD, Rainer F. Storb, MD, Wendy Leisenring, Michael Pulsipher, MD, Michael R. Loken, PhD, Troy Torgerson, MD, Hans D. Ochs, MD, Ann Woolfrey, MD

Blood (2005) 106 (11): 327.

https://doi.org/10.1182/blood.V106.11.327.327

Published: 2005

Journal Articles

Immunomodulation of Transgene Responses Following Naked DNA Transfer of Factor VIII into Hemophilia A Mice.

Peiqing Ye, MD, David J. Rawlings, MD, Arthur R. Thompson, MD, PhD, Hans D. Ochs, MD,Dr.Med, Carol H. Miao, PhD

Blood (2005) 106 (11): 1279.

https://doi.org/10.1182/blood.V106.11.1279.1279

Published: 2005

Journal Articles

WAVE/Scars in platelets

Atsushi Oda, Hiroaki Miki, Ikuo Wada, Hideki Yamaguchi, Daisuke Yamazaki, Shiro Suetsugu, Mineba Nakajima, Akira Nakayama, Katsuya Okawa, Hiroshi Miyazaki, Kazuhiko Matsuno, Hans D. Ochs, Laura M. Machesky, Hiroyoshi Fujita, Tadaomi Takenawa

Blood (2005) 105 (8): 3141–3148.

https://doi.org/10.1182/blood-2003-04-1319

Published: 2005

Journal Articles

Molecular analysis of a large cohort of patients with the hyper immunoglobulin M (IgM) syndrome

Wen-I Lee, Troy R. Torgerson, Michael J. Schumacher, Leman Yel, Qili Zhu, Hans D. Ochs

Blood (2005) 105 (5): 1881–1890.

https://doi.org/10.1182/blood-2003-12-4420

Published: 2005

NARROW

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

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

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

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

Accumulation of peripheral autoreactive B cells in the absence of functional human regulatory T cells

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

Anti-CD3 antibodies modulate anti–factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy

CD4 + FOXP3 + regulatory T cells confer long-term regulation of factor VIII–specific immune responses in plasmid-mediated gene therapy–treated hemophilia mice

Relevance of biallelic versus monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes

Clinical Phenotype and Long Term Outcome in a Large Cohort of X-Linked Thrombocytopenia (XLT)/Mild Wiskott-Aldrich-Syndrome Patients

WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function

Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice

Transient Blockade of ICOS Co-Stimulatory Pathways Induces Long-Term Tolerance to Factor VIII Following Nonviral Gene Transfer of Factor VIII Plasmid into Hemophilia A Mice.

Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice

Postgrafting Immune Suppression Combined with Nonmyeloablative Conditioning for Transplantation of HLA-Identical Hematopoietic Cell Grafts: Results of a Phase I Study for Treatment of Immunodeficiency Disorders.

Immunomodulation of Transgene Responses Following Naked DNA Transfer of Factor VIII into Hemophilia A Mice.

WAVE/Scars in platelets

Molecular analysis of a large cohort of patients with the hyper immunoglobulin M (IgM) syndrome

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American Society of Hematology

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American Society of Hematology

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