HUMAN IMMUNODEFICIENCY VIRUS TYPE I AS A TARGET FOR GENE THERAPY

Magnús Gottfredsson and Paul R. Bohjanen

Division of Infectious Diseases and International Health, Department of Medicine, Duke University Medical Center, Durham, NC 27710

Received 11/17/97 Accepted 11/24/97

1. ABSTRACT

Recent progress in our understanding of the human immunodeficiency virus type 1 (HIV-1) life cycle has lead to the identification and characterization of viral genes or gene products that have been evaluated as targets for gene therapy. Virtually every stage in the viral life cycle and every viral gene product is a potential target. Gene therapy approaches directed at several of these viral targets have been successful at inhibiting HIV-1 replication in cultured human cells, but clinical trials involving gene therapy directed at HIV-1 are still in their infancy. This manuscript begins with a brief review of the viral life cycle with an emphasis on the function of viral gene products and then summarizes the gene therapy approaches that have targeted these viral genes or gene products to inhibit HIV-1 replication.