Lentiviral transgene vectors

Lentiviral transgene vectors

Transgenic animals can be broadly defined as animals that contain recombinant DNA molecules in their genome that have been introduced by human intervention (Wall, 1996). Developmental studies and clinically relevant disease models are based on transgenic laboratory animals, most commonly the mouse. In farm animals, transgenic technology is used to produce large quantities of pharmaceutically re...

Beta thalassemia gene therapy using lentiviral vectors

Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...

Biosafety of lentiviral vectors.

The characteristics of lentiviral vectors (stable integration in non-dividing and dividing cells, long-term expression of the transgene, absence of immune response) make them ideal gene transfer vehicula for future gene therapy. However, the most potent lentiviral vectors are derived from highly pathogenic human viruses, such as HIV. We describe how the field has engineered lentivectors with in...

Lentiviral Vectors in Immunotherapy

Designing Lentiviral Gene Vectors

Gene therapy relies on the delivery of therapeutic genes into patients’ cells. The microdevices used to reach the cells and to transfer the gene payload are called gene vectors. Viral packaging machinery is often utilized to generate the particles transporting the cargo genes. Lentiviruses, a subgroup of retroviruses, are highly suitable for remodeling into gene transfer vectors because they of...