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Similar Molecular Mechanisms, But Divergent Phenotypes in DM1 and DM2—Why?

The RNA binding protein, rbFOX1, competes with MBNL in binding to CCUGexp, but not CUGexp repeats, and thereby mitigates DM2.

Important New Review Articles on DM

The review journal, Frontiers in Neurology, is publishing six new articles on myotonic dystrophy.

Microsatellite Expansions Selectively Trigger Intron Retention

Intronic GC-rich microsatellite expansions are common in neurological disorders and act to trigger the intronic retention that underlies disease pathogenesis.

Quantifying Mutant mRNA in DM1 and DM2

Methodology is reported for robust and reliable quantification of mutant and abberantly spliced RNA in cells from DM patients.

NIH to Release RFA to Re-Compete Rare Disease Clinical Research Network

NIH announces intent to reissue a Funding Opportunity Announcement for the Rare Disease Clinical Research Network competition.

Finding the Right CRISPR Targets for 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?

Interaction of Age and Gender in DM2

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

RAN Translation in DM2

Toxic RNA-mediated mis-splicing and Repeat-Associated Non-AUG (RAN) translation proteins are impacted by free MBNL1 levels and contribute towards CNS pathogenesis in DM2.

Modifying Gene Editing Technology for DM

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. They have modified the Cas9 enzyme so it is targeted to toxic RNA, instead of the expanded DNA repeats in these diseases.

New Drosophila Models for DM1 and DM2

New fly models show a DM2 phenotype at least as severe as DM1 and may provide a platform for studies of genetic modifiers and candidate therapy screening in DM.

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