[HTML][HTML] Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome
L Sereni, MC Castiello, D Di Silvestre… - Journal of Allergy and …, 2019 - Elsevier
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 reconstitution. However, despite the reduced severity and
frequency of bleeding events, platelet counts remain low in GT-treated patients. Objective
We carefully investigated platelet defects in terms of phenotype and function in untreated …
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 reconstitution. However, despite the reduced severity and
frequency of bleeding events, platelet counts remain low in GT-treated patients. Objective
We carefully investigated platelet defects in terms of phenotype and function in untreated …
