Wednesday, March 14, 2012

Fun Fact: RNA Drug Targets for Muscular Dystrophy

For the first time, a series of small molecule have been rationally designed to bind to an RNA structure that is known to be involved in muscular dystrophy. The small molecules were found by querying a defective RNA motif against a database of small molecules. The subsequent ‘hits’ were customized and were found to improve protein splicing defects in cell and animal models. These RNA binding compounds may lead to a novel drug treatment for adult onset of muscular dystrophy.

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