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Of Note


Matthew Disney Receives New Grant to Investigate Myotonic Dystrophy and ALS

The Muscular Dystrophy Association (MDA) has awarded $300,000 over three years to Matthew Disney, professor on the Florida campus of The Scripps Research Institute (TSRI). The new funding will support further work in Disney’s laboratory to optimize two novel drug-like compounds for treating ALS and a form of muscular dystrophy called myotonic dystrophy.

There are more than 30 diseases, including myotonic dystrophy type 1 and ALS (amyotrophic lateral sclerosis or Lou Gehrig’s disease), caused by toxic RNA repeats—errors in the way the genetic code is translated into proteins. These diseases are currently incurable, but Disney’s laboratory is working to develop small molecule drug candidates that can precisely attack and neutralize these toxic repeats, potentially removing the source of the disease from affected cells.

“We want to develop strategies that show there is potential to make drugs that target this class of disease-causing RNAs,” said Disney.

Disney added that, when successful, this approach could open up the field of drug discovery by giving scientists new tools to stop not just myotonic dystrophy type 1 and ALS, but also fragile X syndrome, Huntington’s disease and various ataxias, including spinocerebellar ataxia.

For more information on this research, see Matthew Disney’s Biosketch and the Disney Laboratory Website. The MDA has also published a Q&A with Disney about the work.





Send comments to: press[at]scripps.edu

disney
Professor Matthew Disney and his group on the Florida campus of The Scripps Research Institute hope to open up the field of drug discovery by developing small molecules to target toxic RNA repeats. (Photo by James McEntee.)