Lentiviral gene therapy corrects platelet phenotype and function in Wiskott-Aldrich patients
Academic Article
Publication Date:
2019
abstract:
Thrombocytopenia is a serious issue for all classical Wiskott-Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) patients as it causes severe and life-threatening bleedings. Lentiviral gene therapy (GT) for WAS has shown promising results in terms of immune reconstitution. However, despite the reduced severity and frequency of bleeding events, platelet (PLT) counts remain low in GT -treated patients.
Iris type:
1.1 Articolo in rivista
Keywords:
Gene Therapy; Platelets; Wiskott-Aldrich Syndrome; XLT
List of contributors:
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
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