Ontology highlight
ABSTRACT:
SUBMITTER: Sereni L
PROVIDER: S-EPMC6721834 | biostudies-literature | 2019 Sep
REPOSITORIES: biostudies-literature
Sereni Lucia L Castiello Maria Carmina MC Di Silvestre Dario D Della Valle Patrizia P Brombin Chiara C Ferrua Francesca F Cicalese Maria Pia MP Pozzi Loris L Migliavacca Maddalena M Bernardo Maria Ester ME Pignata Claudio C Farah Roula R Notarangelo Lucia Dora LD Marcus Nufar N Cattaneo Lorella L Spinelli Marco M Giannelli Stefania S Bosticardo Marita M van Rossem Koen K D'Angelo Armando A Aiuti Alessandro A Mauri Pierluigi P Villa Anna A
The Journal of allergy and clinical immunology 20190327 3
<h4>Background</h4>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.<h4>Objective</h4>We carefully investigated platelet defects in term ...[more]