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).
Researchers have identified a compound that helps cells produce more full-length SMN protein from the backup SMN2 gene.
posted on November 6, 2009 - 1:19pm
Scientists have identified a chemical cousin of the commonly used antibiotic tetracycline that has the potential to be refined and modified into a therapy for spinal muscular atrophy (SMA).
PTK-SMA1 works by correcting an error in a cellular process called RNA splicing, and leads to increased production of a critical protein that is deficient in this disease.