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Gene therapy for the treatment of AIDS: animal models and human clinical experience.


Front Biosci. 1999 May 15;4:D468-75. Unique Identifier : AIDSLINE

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.

JOURNAL ARTICLE REVIEW REVIEW, TUTORIAL Acquired Immunodeficiency Syndrome/*THERAPY Animal Disease Models, Animal Gene Products, rev/GENETICS/THERAPEUTIC USE *Gene Therapy Gene Transfer Hematopoiesis Human HIV/GENETICS/PATHOGENICITY Macaca mulatta Mice Mice, SCID RNA, Catalytic/THERAPEUTIC USE Stem Cells/CYTOLOGY/METABOLISM SIV/PATHOGENICITY T-Lymphocytes/CYTOLOGY/METABOLISM Thymus Gland/VIROLOGY Transduction, Genetic


Information in this article was accurate in July 30, 1999. The state of the art may have changed since the publication date. This material is designed to support, not replace, the relationship that exists between you and your doctor. Always discuss treatment options with a doctor who specializes in treating HIV.