Molecular Targets of Gene Transfer Therapy for HIV Infection

THE ACQUIRED immunodeficiency syndrome (AIDS), caused by human immunodeficiency virus (HIV), is a chronic disease primarily characterized by a decrease in CD4-positive T lymphocytes. Subsequent immune system suppression and failure lead to frequent infection by opportunistic pathogens and, oftentimes along with neoplasms or other HIV-related pathology, death of the patient. During the last 10 years, considerable progress has been made in the development of treatments for various opportunistic infections. In addition, the use of nucleoside analogs (zidovudine, dideoxyinosine, dideoxycytodine), which interfere with reverse transcription and seem to slow viral spread, has become commonplace. Although these treatments can delay disease progression,1side effects associated with their use, and possibly the development of viral resistance to these drugs,2may inevitably lead to treatment failure. For these reasons, alternate therapies for HIV infection are needed. Included among the list of potential treatments is gene transfer therapy. Recent progress in ongoing clinical