Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

Stable gene replacement by in vivo administration of lentiviral vectors (LVs) has therapeutic potential for metabolic disorders and other systemic diseases. We studied the expression of intracellular and secreted proteins by LVs in immunocompetent mice. Liver, spleen, and bone marrow cells were effi...

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Veröffentlicht in:	Blood 2004-05, Vol.103 (10), p.3700-3709
Hauptverfasser:	Follenzi, Antonia, Battaglia, Manuela, Lombardo, Angelo, Annoni, Andrea, Roncarolo, Maria Grazia, Naldini, Luigi
Format:	Artikel
Sprache:	eng
Schlagworte:	Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Animals Applied cell therapy and gene therapy Biological and medical sciences Factor IX - administration & dosage Factor IX - genetics Factor IX - immunology Genetic Therapy - methods Genetic Vectors - therapeutic use Green Fluorescent Proteins Hepatocytes - immunology Hepatocytes - metabolism Humans Isoantibodies Lentivirus - genetics Liver - immunology Luminescent Proteins Medical sciences Mice Mice, Transgenic Promoter Regions, Genetic T-Lymphocytes, Cytotoxic - immunology Transduction, Genetic Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transgenes - immunology
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