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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mosby 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6721834/
https://www.ncbi.nlm.nih.gov/pubmed/30926529
http://dx.doi.org/10.1016/j.jaci.2019.03.012

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