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

ABSTRACT:

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 patients with WAS and assessed the effect of GT treatment on platelet dysfunction.

Methods

We analyzed a cohort of 20 patients with WAS/XLT, 15 of them receiving GT. Platelet phenotype and function were analyzed by using electron microscopy, flow cytom

SUBMITTER: Sereni L 

PROVIDER: S-EPMC6721834 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature