Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis

Wiskott-Aldrich-Syndrome (WAS) is a rare X-linked recessive disease caused by mutations of the WAS gene. It is characterized by immunodeficiency, autoimmunity, low numbers of small platelets (microthrombocytopenia) and a high risk of cancer, especially B cell lymphoma and leukemia.

Detalles Bibliográficos
Autores principales: Braun, Christian Joerg, Witzel, Maximilian, Paruzynski, Anna, Boztug, Kaan, von Kalle, Christof, Schmidt, Manfred, Klein, Christoph
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Taylor & Francis 2014
Materias:
Addendum
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4755244/
https://www.ncbi.nlm.nih.gov/pubmed/26942098
http://dx.doi.org/10.4161/21675511.2014.947749
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author Braun, Christian Joerg
Witzel, Maximilian
Paruzynski, Anna
Boztug, Kaan
von Kalle, Christof
Schmidt, Manfred
Klein, Christoph
author_facet Braun, Christian Joerg
Witzel, Maximilian
Paruzynski, Anna
Boztug, Kaan
von Kalle, Christof
Schmidt, Manfred
Klein, Christoph
author_sort Braun, Christian Joerg
collection PubMed
description Wiskott-Aldrich-Syndrome (WAS) is a rare X-linked recessive disease caused by mutations of the WAS gene. It is characterized by immunodeficiency, autoimmunity, low numbers of small platelets (microthrombocytopenia) and a high risk of cancer, especially B cell lymphoma and leukemia.
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spelling pubmed-47552442016-03-03 Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis Braun, Christian Joerg Witzel, Maximilian Paruzynski, Anna Boztug, Kaan von Kalle, Christof Schmidt, Manfred Klein, Christoph Rare Dis Addendum Wiskott-Aldrich-Syndrome (WAS) is a rare X-linked recessive disease caused by mutations of the WAS gene. It is characterized by immunodeficiency, autoimmunity, low numbers of small platelets (microthrombocytopenia) and a high risk of cancer, especially B cell lymphoma and leukemia. Taylor & Francis 2014-10-30 /pmc/articles/PMC4755244/ /pubmed/26942098 http://dx.doi.org/10.4161/21675511.2014.947749 Text en © 2014 Taylor & Francis, LLC http://creativecommons.org/licenses/by-nc/3.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution-Non-Commercial License http://creativecommons.org/licenses/by-nc/3.0/, which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. The moral rights of the named author(s) have been asserted.
spellingShingle Addendum
Braun, Christian Joerg
Witzel, Maximilian
Paruzynski, Anna
Boztug, Kaan
von Kalle, Christof
Schmidt, Manfred
Klein, Christoph
Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
title Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
title_full Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
title_fullStr Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
title_full_unstemmed Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
title_short Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
title_sort gene therapy for wiskott-aldrich syndrome—long-term reconstitution and clinical benefits, but increased risk for leukemogenesis
topic Addendum
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4755244/
https://www.ncbi.nlm.nih.gov/pubmed/26942098
http://dx.doi.org/10.4161/21675511.2014.947749
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