IMR Press / FBL / Volume 4 / Issue 4 / DOI: 10.2741/amado

Frontiers in Bioscience-Landmark (FBL) is published by IMR Press from Volume 26 Issue 5 (2021). Previous articles were published by another publisher on a subscription basis, and they are hosted by IMR Press on as a courtesy and upon agreement with Frontiers in Bioscience.

Gene therapy for the treatment of AIDS: animal models and human clinical experience
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1 Division of Hematology/Oncology,UCLA School of Medicine, 11-964 Factor Building, Los Angeles, CA 90095, USA
2 Department of Microbiology & Molecular Genetics, UCLA School of Medicine, 11-964 Factor Building, Los Angeles, CA 90095, USA
Front. Biosci. (Landmark Ed) 1999, 4(4), 468–475;
Published: 15 May 1999

Although antiretroviral drug therapy has had a significant impact on the natural history of HIV infection, complete virus eradication still remains an unattainable goal. Drug-mediated virological control only occurs transiently, in part as a result of the development of drug resistance. Gene therapy for the treatment of AIDS is a promising area of research that has as its goal the replacement of the HIV-infected cellular pool with cells engineered to resist virus replication. A variety of anti-HIV genes have been designed and tested in laboratory systems, and available results from pilot clinical trials demonstrate the safety and feasibility of this approach. Obstacles to effective application of this technology include partial protection of HIV resistance genes, lack of effective vectoring systems, and unregulated gene expression. Herein, we review recent advances in transduction methods, data from in vivo preclinical studies in relevant animal models, and emerging results derived from pilot clinical gene therapy studies.

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