Lentiviral vectors: key challenges and new developments

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 ...

Lentiviral Gene Therapy Vectors: Challenges and Future Directions

Lentiviral vectors (LV) are efficient vehicles for gene transfer in mammalian cells due to their capacity to stably express a gene of interest in non-dividing and dividing cells. Their use has exponentially grown in the last years both in research and in gene therapy protocols, reaching 12% of the viral vector based clinical trials in 2011 [1]. This chapter reviews and discusses the state of th...

Lentiviral vectors and gene therapy.

Gene therapy is a novel method under investigation for the treatment of genetic, metabolic and neurologic diseases, cancer and AIDS. The primary goal of gene therapy is to deliver a specific gene to a pre-determined target cell, and to direct expression of such a gene in a manner which will result in a therapeutic effect. Retroviral vectors have the ability to integrate in the host cell DNA irr...

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