Azidothymidine (AZT), Reverse Transcriptase Inhibitor

Biological Activity:

Azidothymidine (AZT) is a selective, orally bioavailable and brain penetrant reverse transcriptase inhibitor. It has 100-fold selectivity for HIV reverse transcriptase over DNA polymerase α. It can suppress HIV-1 replication and enhance cell viability in a HIV-1 infected T cell line. It can also suppress growth of a multiple myeloma (MM) cell line and reduces the growth of MM tumor xenografts in mice. In recent studies, AZT can enhance CRISPR-mediated sequence-specific gene knockout via NHEJ in human induced pluripotent stem cells (iPSCs) and other cell types.

How to Use:

In vitro: Azidothymidine (AZT) was used at 5-30 µM final concentration in various in vitro assays.
In vivo: Azidothymidine (AZT) was dosed to mice at 10-50 mg/kg orally once per day.

Reference:

• 1. Mitsuya H, et al. 3'-Azido-3'-deoxythymidine (BW A509U): an antiviral agent that inhibits the infectivity and cytopathic effect of human T-lymphotropic virus type III/lymphadenopathy-associated virus in vitro. (1985) Proc Natl Acad Sci USA. 82(20):7096-100.
• 2. Furman PA, et al. Phosphorylation of 3'-azido-3'-deoxythymidine and selective interaction of the 5'-triphosphate with human immunodeficiency virus reverse transcriptase. (1986) Proc Natl Acad Sci USA. 83(21):8333-7.
• 3. Broder S, et al. The development of antiretroviral therapy and its impact on the HIV-1/AIDS pandemic. (2010) Antiviral Res. 85(1):1-18. 
• 4. Pereira J, et al. Azidothymidine is effective against human multiple myeloma: a new use for an old drug? (2013) Anticancer Agents Med Chem. 13(1):186-92.
• 5. Yu C, et al. Small Molecules Enhance CRISPR Genome Editing in Pluripotent Stem Cells. (2015) Cell Stem Cell 16(2):142-7.