title: Creating genetic resistance to HIV.
abstract: HIV/AIDS remains a chronic and incurable disease, in spite of the notable successes of combination antiretroviral therapy. Gene therapy offers the prospect of creating genetic resistance to HIV that supplants the need for antiviral drugs. In sight of this goal, a variety of anti-HIV genes have reached clinical testing, including gene-editing enzymes, protein-based inhibitors, and RNA-based therapeutics. Combinations of therapeutic genes against viral and host targets are designed to improve the overall antiviral potency and reduce the likelihood of viral resistance. In cell-based therapies, therapeutic genes are expressed in gene modified T lymphocytes or in hematopoietic stem cells that generate an HIV-resistant immune system. Such strategies must promote the selective proliferation of the transplanted cells and the prolonged expression of therapeutic genes. This review focuses on the current advances and limitations in genetic therapies against HIV, including the status of several recent and ongoing clinical studies.
authors.affiliation: Beckman Research Institute of the City of Hope, Duarte, CA, USA.
authors.forename: John C
authors.initials: JC
authors.lastname: Burnett
authors.forename: John A
authors.initials: JA
authors.lastname: Zaia
authors.forename: John J
authors.initials: JJ
authors.lastname: Rossi
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000188:drug therapy / Q000235:genetics / Q000276:immunology; D019380:Anti-HIV Agents / Q000191:economics / Q000627:therapeutic use; D044966:Anti-Retroviral Agents / Q000191:economics / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D024882:Drug Resistance, Viral / Q000235:genetics* / Q000276:immunology; D015316:Genetic Therapy / Q000191:economics / Q000379:methods*; D015497:HIV-1 / Q000187:drug effects / Q000235:genetics* / Q000276:immunology; D006801:Humans
chemical_list: D019380:Anti-HIV Agents; D044966:Anti-Retroviral Agents