DM1

Development of a mini gene tool facilitates the identification of candidate therapeutics targeted at dissociating MBNL from expanded CUG repeats.

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?

Genetic and epigenetic mechanisms drive differences between CDM and DM1—a new study elucidates downstream signaling pathways that underlie their diverse phenotypes and represent putative therapy development targets for CDM.

Age and gender impact the onset and progression of DM2, but the pattern shows both similarities and differences from that of DM1.

It would be easy to conclude that insulin resistance and diabetes in myotonic dystrophy is a product of mis-splicing of INSR transcripts—but is that the full story?

Investigators at the University of California San Diego, the University of Florida, and the National University of Singapore have recently reported early research that potentially ‘repurposes’ gene editing technology for a set of RNA disorders—myotonic dystrophy type 1 (DM1), myotonic dystrophy type 2 (DM2), a subset of Lou Gehrig’s disease (ALS) patients and Huntington’s disease.