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Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome

BACKGROUND: Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe and life-threatening bleeding. Lentiviral gene therapy (GT) for WAS has shown promising results in terms of immune reconstitutio...

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Библиографические подробности
Опубликовано в: :J Allergy Clin Immunol
Главные авторы: Sereni, Lucia, Castiello, Maria Carmina, Di Silvestre, Dario, Della Valle, Patrizia, Brombin, Chiara, Ferrua, Francesca, Cicalese, Maria Pia, Pozzi, Loris, Migliavacca, Maddalena, Bernardo, Maria Ester, Pignata, Claudio, Farah, Roula, Notarangelo, Lucia Dora, Marcus, Nufar, Cattaneo, Lorella, Spinelli, Marco, Giannelli, Stefania, Bosticardo, Marita, van Rossem, Koen, D'Angelo, Armando, Aiuti, Alessandro, Mauri, Pierluigi, Villa, Anna
Формат: Artigo
Язык:Inglês
Опубликовано: Mosby 2019
Предметы:
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6721834/
https://ncbi.nlm.nih.gov/pubmed/30926529
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jaci.2019.03.012
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