Intronic GC-rich microsatellite expansions are common in neurological disorders and act to trigger the intronic retention that underlies disease pathogenesis.
Methodology is reported for robust and reliable quantification of mutant and abberantly spliced RNA in cells from DM patients.
NIH announces intent to reissue a Funding Opportunity Announcement for the Rare Disease Clinical Research Network competition.
CRISPR is an exploratory strategy with potential for treatment of RNA-triggered diseases. Will DNA or RNA targeting prove to be the best approach for DM?
Age and gender impact the onset and progression of DM2, but the pattern shows both similarities and differences from that of DM1.