Enzyme Responsible For Muscular Dystrophy Uncovered

by VR Sreeraman on Dec 9 2008 4:10 PM

Scientists at an American research institute claim that they have been successful in reversing the impact of a mutation in the gene coding the protein dystrophin, known to be associated with muscular dystrophy.

Dr. Lorenzo Puri and his colleagues at the Burnham Institute for Medical Research say that their study showed that the dystrophin mutation causes an increase in the amount of the histone deacetylase enzyme, HDAC2, which in turn alters the gene expression profile in the diseased muscle cells compared to normal muscle cells.

The researchers say that inhibiting HDAC2 with small molecule compounds or RNA interference helped return the muscle cells with the dystrophin mutation to normal histology and function.

They claim that their breakthrough work might lead to novel therapies for muscular dystrophy.