A new study examines the role of the GSK3β—cyclin D3—CUGBP1 pathway in the pathogenesis of DM1 and its potential as a therapeutic target.
Development of a mini gene tool facilitates the identification of candidate therapeutics targeted at dissociating MBNL from expanded CUG repeats.
Increased skin cancer risks in DM1 are captured in a new, longitudinal electronic medical records study.
A recent publication suggests that a serum protein may ultimately have value as a biomarker for conduction system abnormalities in DM.
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?