RNA-mediated inhibition of HIV in a gene therapy setting.
At present, treatment for HIV-1 infection employs highly active anti-retroviral therapy (HAART), which utilizes a combination of RT and protease inhibitors. Unfortunately, HIV can escape many therapies because of its high mutation rate and the complexity of its pathogenesis. HIV-1 integrates into the cellular genome, which facilitates persistence and acts as a reservoir for reactivation and replication. As an alternative or adjuvant to chemotherapy we have been developing an RNA-based gene therapy approach for the treatment of HIV-1 infection. This article summarizes the various RNA based technologies that we have developed for potential application in a gene therapy setting.
Michienzi
Alessandro
A
Divisions of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, California 91010, USA.
Castanotto
Daniela
D
Lee
Nancy
N
Li
Shirley
S
Zaia
John A
JA
Rossi
John J
JJ
D019380:Anti-HIV Agents / Q000494:pharmacology*; D002466:Cell Nucleolus / Q000187:drug effects; D015316:Genetic Therapy / Q000379:methods*; D006678:HIV / Q000187:drug effects*; D015658:HIV Infections / Q000188:drug therapy*; D006412:Hematopoietic Stem Cells; D006801:Humans; D034622:RNA Interference / Q000187:drug effects; D016337:RNA, Catalytic / Q000494:pharmacology*; D034741:RNA, Small Interfering / Q000187:drug effects
D019380:Anti-HIV Agents; D016337:RNA, Catalytic; D034741:RNA, Small Interfering