Abstract

Clinical application of gene transfer technology continues to be investigated as a possible future therapeutic modality for a variety of human diseases. This article discusses the basic characteristics of human immunodeficiency virus type 1 (HIV-1) that have made it both an attractive target for possible gene therapy intervention and a potential tool for use in a variety of other clinical gene transfer applications. The molecular genetics and features of the replication cycle of HIV-1 relevant to gene therapy are discussed. Fundamental strategies for using gene transfer as an anti-HIV-1 therapy are reviewed, using examples of specific targets and methodology to illustrate each concept. The rationale for development and components of HIV-1 vector systems for use in the clinical gene therapy are discussed.

Keywords: human Immunodeficiency virus (hiv), gene therapy, lentivirus, viral vector, replication, ribozyme, safety, retrovirus