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Azacitidine

Abstract

Azacitidine (Vidaza; Pharmion), an inhibitor of DNA methylation, was approved by the US FDA for the treatment of myelodysplastic syndromes in May 2004. It is the first drug to be approved by the FDA for treating this rare family of bone-marrow disorders, and has been given orphan-drug status. It is also a pioneering example of an agent that targets 'epigenetic' gene silencing, a mechanism that is exploited by cancer cells to inhibit the expression of genes that counteract the malignant phenotype.

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Figure 1: Azacitidine and DNA methylation.

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Correspondence to Jean-Pierre J. Issa or Peter Kirkpatrick.

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Issa, JP., Kantarjian, H. & Kirkpatrick, P. Azacitidine. Nat Rev Drug Discov 4, 275–276 (2005). https://doi.org/10.1038/nrd1698

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