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Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott–Aldrich syndrome
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Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott–Aldrich syndrome
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Journal Article
Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott–Aldrich syndrome
2012
Overview
Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency caused by the defective expression of the WAS protein (WASP) in hematopoietic cells. It has been shown that dendritic cells (DCs) are functionally impaired in WAS patients and
was
−/−
mice. We have previously demonstrated the efficacy and safety of a murine model of WAS gene therapy (GT), using stem cells transduced with a lentiviral vector (LV). The aim of this study was to investigate whether GT can correct DC defects in
was
−/−
mice. As DCs expressing WASP were detected in the secondary lymphoid organs of the treated mice, we tested the
in vitro
and
in vivo
function of bone marrow-derived DCs (BMDCs). The BMDCs showed efficient
in vitro
uptake of latex beads and
Salmonella typhimurium
. When BMDCs from the treated mice (GT BMDCs) and the
was
−/−
mice were injected into wild-type hosts, we found a higher number of cells that had migrated to the draining lymph nodes compared with mice injected with
was
−/−
BMDCs. Finally, we found that ovalbumin (OVA)-pulsed GT BMDCs or vaccination of GT mice with anti-DEC205 OVA fusion protein can efficiently induce antigen-specific T-cell activation
in vivo
. These findings show that WAS GT significantly improves DC function, thus adding new evidence of the preclinical efficacy of LV-mediated WAS GT.
Publisher
Nature Publishing Group UK,Nature Publishing Group
Subject
/ Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy
/ Animals
/ Applied cell therapy and gene therapy
/ Biological and medical sciences
/ Biomedical and Life Sciences
/ Bone Marrow Cells - immunology
/ Dendritic Cells - immunology
/ Dendritic Cells - metabolism
/ Fundamental and applied biological sciences. Psychology
/ Health. Pharmaceutical industry
/ Humans
/ Industrial applications and implications. Economical aspects
/ Lymphoid Tissue - metabolism
/ Mice
/ Proteins
/ Transfusions. Complications. Transfusion reactions. Cell and gene therapy
/ Wiskott-Aldrich Syndrome - therapy
/ Wiskott-Aldrich Syndrome Protein - genetics
