AIDS and HIV infection.

Abstract

Infection with the human immunodeficiency virus (HIV) is a paradigm for serious viral diseases which may be treatable with gene therapy. The biology of the virus and the pathophysiology of infection with HIV are rapidly being clarified and a number of potential targets for gene therapy interventions have been identified. HIV infects primarily the hematopoietic system which is relatively easy to obtain, manipulate, assess and transplant. Current pharmacologic, immunologic, and biotherapeutic approaches to treating HIV infection have been disappointing, propelling the development of alternative treatment approaches. This chapter will review the features of HIV biology relevant to gene therapy, HIV gene therapy inhibition strategies and gene delivery systems, preclinical models for evaluating the safety and efficacy of gene therapy approaches to HIV infection, and the initial potential clinical applications of this technology.