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Small molecules that induce or reverse effects of the type 1 myotonic dystrophy mutation in cells in the lab are likely to speed research

posted on July 9, 2013 - 11:01am
Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).

Compound frees a crucial protein from a cellular trap in mice with MMD1; treatment prospects 'bright'

posted on July 17, 2009 - 4:03pm
Researchers at the University of Rochester (N.Y.) Wellstone Muscular Dystrophy Cooperative Research Center have identified a compound that has the potential to be developed into a treatment for type 1 myotonic dystrophy (MMD1, or DM1). The compound, dubbed CAG25, is an "antisense oligonucleotide," a type of construct that's been used to block disease-causing genetic instructions in laboratory...