Compositions for and uses thereof for altering gene expression with phenylacetate and derivatives thereof

摘要

A pharmaceutical composition for inhibiting altering gene expression, inducing differentiation in nonmalignant mammalian cells, and for the treatment of beta -chain hemoglobinopathies, comprises a pharmaceutically effective amount of a compound of formula I: R<1>R<2>CH-COOH wherein R<1> is selected from H, CH3, CH3-O-, C2H5 or C3H7; R<2> is selected from X is wherein X is independently selected from halogen or hydroxy, and n is 0 to 4; a stereoisomer thereof, or a pharmaceutically acceptable salt thereof, and mixtures thereof together with a pharmaceutically acceptable carrier.