[HTML][HTML] B-cell reconstitution after lentiviral vector–mediated gene therapy in patients with Wiskott-Aldrich syndrome

MC Castiello, S Scaramuzza, F Pala, F Ferrua… - Journal of Allergy and …, 2015 - Elsevier
Background Wiskott-Aldrich syndrome (WAS) is a severe X-linked immunodeficiency
characterized by microthrombocytopenia, eczema, recurrent infections, and susceptibility to
autoimmunity and lymphomas. Hematopoietic stem cell transplantation is the treatment of
choice; however, administration of WAS gene–corrected autologous hematopoietic stem
cells has been demonstrated as a feasible alternative therapeutic approach. Objective
Because B-cell homeostasis is perturbed in patients with WAS and restoration of immune …