1. ¹Department of Pathology, Jefferson Medical College, 1020 Locust Street, Room 251, Philadelphia, PA 19107, USA
2. ²Department of Microbiology, Immunology and Pathology, Colorado State University, Fort Collins, CO, USA
3. ³Center for Gene Therapy, Department of Pharmacology, Tulane University Health Sciences Center, New Orleans, LA, USA
4. ⁴Lentigen Corp., Clarksville, MD, USA
5. ⁵Department of Pathology, University of Utah School of Medicine, Salt Lake City, UT, USA
6. ⁶Department of Medicine, Jefferson Medical College, Philadelphia, PA 19107, USA
7. ⁷Department of Molecular Biology, Duarte, CA, USA
8. ⁸Department of Virology, Beckman Research Institute, City of Hope Medical Center, Duarte, CA, USA

Correspondence: David S. Strayer, Department of Pathology, Jefferson Medical College, 1020 Locust Street, Room 251, Philadelphia, PA 19107, USA. Fax: (215) 503-1156. E-mail: david.strayer@jefferson.edu

Received 24 August 2004; Accepted 26 January 2005.

Abstract

Progress in developing effective gene transfer approaches to treat HIV-1 infection has been steady. Many different transgenes have been reported to inhibit HIV-1 in vitro. However, effective translation of such results to clinical practice, or even to animal models of AIDS, has been challenging. Among the reasons for this failure are uncertainty as to the most effective cell population(s) to target, the diffuseness of these target cells in the body, and ineffective or insufficiently durable gene delivery. Better understanding of the HIV-1 replicative cycle, host factors involved in HIV-1 infection, vector biology and application, transgene technology, animal models, and clinical study design have all contributed vastly to planning current and future strategies for application of gene therapeutic approaches to the treatment of AIDS. This review focuses on the newest developments in these areas and provides a strong basis for renewed optimism that gene therapy will have an important role to play in treating people infected with HIV-1.