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.
Lentiviral gene therapy corrects platelet phenotype and function in Wiskott-Aldrich patients
Brombin, Chiara;Cicalese, Maria Pia;Bernardo, Maria Ester;Aiuti, Alessandro;
2019-01-01
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.File in questo prodotto:
Non ci sono file associati a questo prodotto.
I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
