Azacitidine

JPJ Issa, HM Kantarjian, P Kirkpatrick - Nature reviews. Drug discovery, 2005 - nature.com
JPJ Issa, HM Kantarjian, P Kirkpatrick
Nature reviews. Drug discovery, 2005nature.com
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.
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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