Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome
M Bosticardo, E Draghici, F Schena, AV Sauer… - Journal of allergy and …, 2011 - Elsevier
Background Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency
characterized by thrombocytopenia, eczema, infections, autoimmunity, and lymphomas.
Transplantation of hematopoietic stem cells from HLA-identical donors is curative, but it is
not available to all patients. We have developed a gene therapy (GT) approach for WAS by
using a lentiviral vector encoding for human WAS promoter/cDNA (w1. 6W) and
demonstrated its preclinical efficacy and safety. Objective To evaluate B-cell reconstitution …
characterized by thrombocytopenia, eczema, infections, autoimmunity, and lymphomas.
Transplantation of hematopoietic stem cells from HLA-identical donors is curative, but it is
not available to all patients. We have developed a gene therapy (GT) approach for WAS by
using a lentiviral vector encoding for human WAS promoter/cDNA (w1. 6W) and
demonstrated its preclinical efficacy and safety. Objective To evaluate B-cell reconstitution …
