Switching Off Muscular Dystrophy

A group of compounds can switch off the genes responsible for adult-onset muscular dystrophy. This is one of the first times scientists have been able to turn off a genetic, hereditary disease. Chemists screened more than 300,000 small molecules from a previous NIH-funded study and isolated compounds that bind to and inhibit an RNA complex critical to the disease’s progression. When other scientists on the team introduced the compounds into in vitro muscle tissue affected by the disease, the RNA complex did not form, allowing the damage to the muscle cells to be reversed. The small molecules will serve as a tool for studies on muscular dystrophy and investigations into their use as potential disease treatments.
Source: NIGMS Biomedical Beat - Category: Research Source Type: news